Publications by authors named "William R Lumry"

50 Publications

Long-term health-related quality of life in patients treated with subcutaneous C1-inhibitor replacement therapy for the prevention of hereditary angioedema attacks: findings from the COMPACT open-label extension study.

Orphanet J Rare Dis 2021 Feb 15;16(1):86. Epub 2021 Feb 15.

University College Hospital, London, UK.

Background: Long-term prophylaxis with subcutaneous C1-inhibitor (C1-INH[SC]; HAEGARDA, CSL Behring) in patients with hereditary angioedema (HAE) due to C1-INH deficiency (C1-INH-HAE) was evaluated in an open-label extension follow-up study to the international, double-blind, placebo-controlled COMPACT study. The current analysis evaluated patient-reported health-related quality of life (HRQoL) data from 126 patients in the open-label extension study randomized to treatment with C1-INH(SC) 40 IU/kg (n = 63) or 60 IU/kg (n = 63) twice weekly for 52 weeks. HRQoL was evaluated at the beginning of the open-label study and at various time points using the European Quality of Life-5 Dimensions Questionnaire (EQ-5D), the Hospital Anxiety and Depression Scale (HADS), the Work Productivity and Activity Impairment Questionnaire (WPAI), and the Treatment Satisfaction Questionnaire for Medication. The disease-specific Angioedema Quality of Life Questionnaire (AE-QoL) and HAE quality of life questionnaire (HAE-QoL) instruments were administered in a subset of patients. Statistical significance was determined by change-from-baseline 95% confidence intervals (CIs) excluding zero. No adjustment for multiplicity was done.

Results: Mean baseline EQ-5D scores (Health State Value, 0.90; Visual Analog Scale, 81.32) were slightly higher (better) than United States population norms (0.825, 80.0, respectively) and mean HADS anxiety (5.48) and depression (2.88) scores were within "normal" range (0-7). Yet, patients using C1-INH(SC) 60 IU/kg demonstrated significant improvement from baseline to end-of-study on the EQ-5D Health State Value (mean change [95% CI], 0.07 [0.01, 0.12] and Visual Analog Scale (7.45 [3.29, 11.62]). In the C1-INH(SC) 60 IU/kg group, there were significant improvements in the HADS anxiety scale (mean change [95% CI], - 1.23 [- 2.08, - 0.38]), HADS depression scale (- 0.95 [- 1.57, - 0.34]), and WPAI-assessed presenteeism (mean change [95% CI], - 23.33% [- 34.86, - 11.81]), work productivity loss (- 26.68% [- 39.92, - 13.44]), and activity impairment (- 16.14% [- 26.36, - 5.91]). Clinically important improvements were achieved in ≥ 25% of patients for all domains except WPAI-assessed absenteeism (which was very low at baseline). Mean AE-QoL total score by visit ranged from 13.39 to 17.89 (scale 0-100; lower scores = less impairment). Mean HAE-QoL global scores at each visit (115.7-122.3) were close to the maximum (best) possible score of 135.

Conclusions: Long-term C1-INH(SC) replacement therapy in patients with C1-INH-HAE leads to significant and sustained improvements in multiple measures of HRQoL. Trial registration A Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema, NCT02316353. Registered December 12, 2014, https://clinicaltrials.gov/ct2/show/NCT02316353 .
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http://dx.doi.org/10.1186/s13023-020-01658-4DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7885603PMC
February 2021

Impact of lanadelumab on health-related quality of life in patients with hereditary angioedema in the HELP study.

Allergy 2021 Apr 24;76(4):1188-1198. Epub 2020 Dec 24.

Dermatological Allergology, Allergie-Centrum-Charité, Department of Dermatology and Allergy, Charité - Universitätsmedizin Berlin, Berlin, Germany.

Background: An objective of the phase 3 HELP Study was to investigate the effect of lanadelumab on health-related quality of life (HRQoL) in patients with hereditary angioedema (HAE).

Methods: Patients with HAE-1/2 received either lanadelumab 150 mg every 4 weeks (q4wks; n = 28), 300 mg q4wks (n = 29), 300 mg every 2 weeks (q2wks; n = 27), or placebo (n = 41) for 26 weeks (days 0-182). The Angioedema Quality of Life Questionnaire (AE-QoL) was administered monthly, consisting of four domain (functioning, fatigue/mood, fears/shame, nutrition) and total scores. The generic EQ-5D-5L questionnaire was administered on days 0, 98, and 182. Comparisons were made between placebo and (a) all lanadelumab-treated patients and (b) individual lanadelumab groups for changes in scores (day 0-182) and proportions achieving the minimal clinically important difference (MCID, -6) in AE-QoL total score.

Results: Compared with the placebo group, the lanadelumab total group demonstrated significantly greater improvements in AE-QoL total and domain scores (mean change, -13.0 to -29.3; p < 0.05 for all); the largest improvement was in functioning. A significantly greater proportion of the lanadelumab total group achieved the MCID (70% vs 37%; p = 0.001). The lanadelumab 300 mg q2wks group had the highest proportion (81%; p = 0.001) and was 7.2 times more likely to achieve the MCID than the placebo group. Mean EQ-5D-5L scores at day 0 were high in all groups, indicating low impairment, with no significant changes at day 182.

Conclusion: Patients with HAE-1/2 experienced significant and clinically meaningful improvements in HRQoL measured by AE-QoL following lanadelumab treatment in the HELP Study.
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http://dx.doi.org/10.1111/all.14680DOI Listing
April 2021

Hereditary angioedema: Epidemiology and burden of disease.

Allergy Asthma Proc 2020 11;41(Suppl 1):S08-S13

Warren Alpert Medical School, Brown University, Providence, RI.

Hereditary angioedema (HAE) is a rare autosomal dominant genetic disorder characterized by swelling of subcutaneous, mucosal, and submucosal tissue without associated pruritus or wheals caused by a temporary localized increase in vascular permeability. Swelling attacks primarily affect the cutaneous tissue, abdominal viscera, genitals, or airways. <title/> Reports of the prevalence of HAE C1 inhibitor (C1-INH) deficiency varies widely, from 1:50,000 to 1:100,000. The prevalence of HAE normal C1-INH is unknown but is likely much lower than HAE C1-INH. Approximately one-third of patients with recurrent angioedema without wheals have HAE. <title/> The burden of disease for patients with HAE is substantial. Attacks are unpredictable with respect to frequency, severity, and the site that swells. Laryngeal attacks can be fatal if not treated promptly and appropriately. Feelings of stress, anxiety, and depression can trigger attacks, and begin a cycle of attacks that cause anxiety that, in turn, triggers further attacks. Despite full physical recovery between attacks, patients often experience continual emotional impairment and reduced quality of life (QoL). Absenteeism from work and presenteeism at work or educational activities for patients and caregivers increase stress and reduce productivity during and between attacks. Missed opportunities for career development are common. <title/> Significant advances have been made in the past decade to expand both acute and prophylactic treatment options for patients with HAE, lowering both the disease and treatment burden, and improving the QoL of patients with HAE.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/33109318/Hereditary-angioedema-Epidemiology-and-burden-of-disease" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.2500/aap.2020.41.200050' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-33109318-n' >http://dx.doi.org/10.2500/aap.2020.41.200050</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> November 2020</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/33109318" target="_blank" title="" class="fb socialShare" id="fb-a-33109318"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/33109318" target="_blank" title="" class="tw socialShare" id="tw-a-33109318"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/33109318&title=Hereditary+angioedema%3A+Epidemiology+and+burden+of+disease.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-33109318"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/33098856/Oral-once-daily-berotralstat-for-the-prevention-of-hereditary-angioedema-attacks-Arandomized-double-" >Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: A randomized, double-blind, placebo-controlled phase 3 trial.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Bruce+Zuraw'>Bruce Zuraw</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Douglas+T+Johnston'>Douglas T Johnston</a> <a href='https://www.pubfacts.com/author/Emel+Aygören-Pürsün'>Emel Aygören-Pürsün</a> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/Sandra+C+Christiansen'>Sandra C Christiansen</a> <a href='https://www.pubfacts.com/author/Joshua+S+Jacobs'>Joshua S Jacobs</a> <a href='https://www.pubfacts.com/author/Karl+V+Sitz'>Karl V Sitz</a> <a href='https://www.pubfacts.com/author/Richard+G+Gower'>Richard G Gower</a> <a href='https://www.pubfacts.com/author/Remi+Gagnon'>Remi Gagnon</a> <a href='https://www.pubfacts.com/author/H+James+Wedner'>H James Wedner</a> <a href='https://www.pubfacts.com/author/Tamar+Kinaciyan'>Tamar Kinaciyan</a> <a href='https://www.pubfacts.com/author/Roman+Hakl'>Roman Hakl</a> <a href='https://www.pubfacts.com/author/Jana+Hanzlíková'>Jana Hanzlíková</a> <a href='https://www.pubfacts.com/author/John+T+Anderson'>John T Anderson</a> <a href='https://www.pubfacts.com/author/Donald+L+McNeil'>Donald L McNeil</a> <a href='https://www.pubfacts.com/author/Stephen+B+Fritz'>Stephen B Fritz</a> <a href='https://www.pubfacts.com/author/William+H+Yang'>William H Yang</a> <a href='https://www.pubfacts.com/author/Raffi+Tachdjian'>Raffi Tachdjian</a> <a href='https://www.pubfacts.com/author/Paula+J+Busse'>Paula J Busse</a> <a href='https://www.pubfacts.com/author/Timothy+J+Craig'>Timothy J Craig</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/Henriette+Farkas'>Henriette Farkas</a> <a href='https://www.pubfacts.com/author/Jessica+M+Best'>Jessica M Best</a> <a href='https://www.pubfacts.com/author/Desiree+Clemons'>Desiree Clemons</a> <a href='https://www.pubfacts.com/author/Melanie+Cornpropst'>Melanie Cornpropst</a> <a href='https://www.pubfacts.com/author/Sylvia+M+Dobo'>Sylvia M Dobo</a> <a href='https://www.pubfacts.com/author/Heather+A+Iocca'>Heather A Iocca</a> <a href='https://www.pubfacts.com/author/Deborah+Kargl'>Deborah Kargl</a> <a href='https://www.pubfacts.com/author/Eniko+Nagy'>Eniko Nagy</a> <a href='https://www.pubfacts.com/author/Sharon+C+Murray'>Sharon C Murray</a> <a href='https://www.pubfacts.com/author/Phil+Collis'>Phil Collis</a> <a href='https://www.pubfacts.com/author/William+P+Sheridan'>William P Sheridan</a> <a href='https://www.pubfacts.com/author/Marcus+Maurer'>Marcus Maurer</a> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>J Allergy Clin Immunol 2020 Oct 21. Epub 2020 Oct 21.</p><p>University of California San Diego, San Diego, Calif.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: Berotralstat (BCX7353) is an oral, once-daily inhibitor of plasma kallikrein in development for the prophylaxis of hereditary angioedema (HAE) attacks.<BR><BR><em>Objective</em>: Our aim was to determine the efficacy, safety, and tolerability of berotralstat in patients with HAE over a 24-week treatment period (the phase 3 APeX-2 trial).<BR><BR><em>Methods</em>: APeX-2 was a double-blind, parallel-group study that randomized patients at 40 sites in 11 countries 1:1:1 to receive once-daily berotralstat in a dose of 110 mg or 150 mg or placebo (Clinicaltrials.gov identifier NCT03485911). Patients aged 12 years or older with HAE due to C1 inhibitor deficiency and at least 2 investigator-confirmed HAE attacks in the first 56 days of a prospective run-in period were eligible. The primary efficacy end point was the rate of investigator-confirmed HAE attacks during the 24-week treatment period.<BR><BR><em>Results</em>: A total of 121 patients were randomized; 120 of them received at least 1 dose of the study drug (n = 41, 40, and 39 in the 110-mg dose of berotralstat, 150-mg of dose berotralstat, and placebo groups, respectively). Berotralstat demonstrated a significant reduction in attack rate at both 110 mg (1.65 attacks per month; P = .024) and 150 mg (1.31 attacks per month; P < .001) relative to placebo (2.35 attacks per month). The most frequent treatment-emergent adverse events that occurred more with berotralstat than with placebo were abdominal pain, vomiting, diarrhea, and back pain. No drug-related serious treatment-emergent adverse events occurred.<BR><BR><em>Conclusion</em>: Both the 110-mg and 150-mg doses of berotralstat reduced HAE attack rates compared with placebo and were safe and generally well tolerated. The most favorable benefit-to-risk profile was observed at a dose of 150 mg per day.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/33098856/Oral-once-daily-berotralstat-for-the-prevention-of-hereditary-angioedema-attacks-Arandomized-double-" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.jaci.2020.10.015' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-33098856-n' >http://dx.doi.org/10.1016/j.jaci.2020.10.015</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> October 2020</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/33098856" target="_blank" title="" class="fb socialShare" id="fb-a-33098856"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/33098856" target="_blank" title="" class="tw socialShare" id="tw-a-33098856"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/33098856&title=Oral+once-daily+berotralstat+for+the+prevention+of+hereditary+angioedema+attacks%3A+A%C2%A0randomized%2C+double-blind%2C+placebo-controlled+phase+3+trial.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-33098856"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/32898710/US-HAEA-Medical-Advisory-Board-2020-Guidelines-for-the-Management-of-Hereditary-Angioedema" >US HAEA Medical Advisory Board 2020 Guidelines for the Management of Hereditary Angioedema.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Paula+J+Busse'>Paula J Busse</a> <a href='https://www.pubfacts.com/author/Sandra+C+Christiansen'>Sandra C Christiansen</a> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/Anthony+J+Castaldo'>Anthony J Castaldo</a> <a href='https://www.pubfacts.com/author/Timothy+Craig'>Timothy Craig</a> <a href='https://www.pubfacts.com/author/Mark+Davis-Lorton'>Mark Davis-Lorton</a> <a href='https://www.pubfacts.com/author/Michael+M+Frank'>Michael M Frank</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Bruce+L+Zuraw'>Bruce L Zuraw</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>J Allergy Clin Immunol Pract 2021 Jan 6;9(1):132-150.e3. Epub 2020 Sep 6.</p><p>Division of Rheumatology, Allergy, and Immunology, Department of Medicine, University of California San Diego, La Jolla, Calif; San Diego Veterans Administration Healthcare, San Diego, Calif. Electronic address: </p><div class="pubDescriptor" style="margin-top:20px;">Scientific and clinical progress together with the development of effective novel therapeutic options has engendered multiple important changes in the diagnosis and management of hereditary angioedema (HAE). We now update and extend the 2013 United States Hereditary Angioedema Association Medical Advisory Board guidelines for the treatment and management of HAE. The guidelines are based on a comprehensive literature review with recommendations indicating both the strength of our recommendation and the quality of the underlying evidence. Guidelines are provided regarding the classification, diagnosis, on-demand treatment, prophylactic treatment, special considerations for women and children, development of a comprehensive management and monitoring plan, and assessment of burden of illness for both HAE due to C1 inhibitor deficiency and HAE with normal C1 inhibitor. Advances in HAE treatment now allow the development of management plans that can help many patients with HAE lead a normal life. Achieving this goal requires that physicians be familiar with the diagnostic and therapeutic transformations that have occurred in recent years.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/32898710/US-HAEA-Medical-Advisory-Board-2020-Guidelines-for-the-Management-of-Hereditary-Angioedema" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.jaip.2020.08.046' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-32898710-n' >http://dx.doi.org/10.1016/j.jaip.2020.08.046</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> January 2021</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/32898710" target="_blank" title="" class="fb socialShare" id="fb-a-32898710"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/32898710" target="_blank" title="" class="tw socialShare" id="tw-a-32898710"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/32898710&title=US+HAEA+Medical+Advisory+Board+2020+Guidelines+for+the+Management+of+Hereditary+Angioedema.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-32898710"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/32534150/Association-Between-Self-Reported-Dental-Hygiene-Practices-and-Dental-Procedure-Related-Recurrent-An" >Association Between Self-Reported Dental Hygiene Practices and Dental Procedure-Related Recurrent Angioedema Attacks in HAE Subjects: A Multicenter Survey.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Umesh+Singh'>Umesh Singh</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Paula+Busse'>Paula Busse</a> <a href='https://www.pubfacts.com/author/H+James+Wedner'>H James Wedner</a> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> <a href='https://www.pubfacts.com/author/Timothy+J+Craig'>Timothy J Craig</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/Raffi+Tachdjian'>Raffi Tachdjian</a> <a href='https://www.pubfacts.com/author/Joshua+S+Jacobs'>Joshua S Jacobs</a> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> <a href='https://www.pubfacts.com/author/Mark+Davis-Lorton'>Mark Davis-Lorton</a> <a href='https://www.pubfacts.com/author/Sandra+C+Christiansen'>Sandra C Christiansen</a> <a href='https://www.pubfacts.com/author/Bruce+L+Zuraw'>Bruce L Zuraw</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>J Allergy Clin Immunol Pract 2020 10 10;8(9):3162-3169.e5. Epub 2020 Jun 10.</p><p>Division of Immunology/Allergy, Department of Internal Medicine, University of Cincinnati College of Medicine, Cincinnati, Ohio. Electronic address: </p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: Hereditary angioedema (HAE) symptoms may be triggered by dental procedures, thereby complicating dental care in individuals affected by the condition.<BR><BR><em>Objective</em>: This study investigated the self-perceived dental care needs, perceived susceptibility to acute angioedema (AE) attacks after dental procedures, and dental care behavior of patients with HAE.<BR><BR><em>Methods</em>: A self-administered semistructured web-based questionnaire was distributed to 250 adult patients with HAE (type 1 or 2; 88% type 1) and 256 matched non-HAE controls. Data were analyzed using stratified χ tests, logistic regression, and classification trees.<BR><BR><em>Results</em>: A total of 46.4% of HAE versus 55.5% of control patients had dental visits within 6 months (P = .04). Dental insurance was a barrier to seeking routine dental visits among both groups. However, significantly fewer patients with HAE had routine dental visits within 6 months despite having dental insurance compared with control patients (48% vs 60%, P = .01). Within the HAE group, a significantly greater number of patients with dental visits at intervals greater than 6 months had a history of recurrent postprocedural AE attacks (odds ratio [OR]: 3.9 [1.7, 8.8], P = .0005) and used antibacterial toothpaste more frequently than those without recurrent AE attacks (OR: 4.7 [1.5, 15.4], P = .005).<BR><BR><em>Conclusions</em>: These data support the hypothesis that patients with HAE who are predisposed to having AE episodes in response to medical or physical trauma visit the dentist less and engage in specific oral hygiene practices more frequently than matched control patients and patients with HAE who reported that they were less likely to swell after a dental procedure.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/32534150/Association-Between-Self-Reported-Dental-Hygiene-Practices-and-Dental-Procedure-Related-Recurrent-An" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.jaip.2020.05.041' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-32534150-n' >http://dx.doi.org/10.1016/j.jaip.2020.05.041</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> October 2020</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/32534150" target="_blank" title="" class="fb socialShare" id="fb-a-32534150"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/32534150" target="_blank" title="" class="tw socialShare" id="tw-a-32534150"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/32534150&title=Association+Between+Self-Reported+Dental+Hygiene+Practices+and+Dental+Procedure-Related+Recurrent+Angioedema+Attacks+in+HAE+Subjects%3A+A+Multicenter+Survey.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-32534150"><i class="icon-linkedin"></i></a> </div></div> </div> </div> <div class="row" > <div class="col-md-12" style="text-align:center;" ><script async src="https://pagead2.googlesyndication.com/pagead/js/adsbygoogle.js"></script> <!-- 2) Display - Horizontal - Author - Responsive --> <ins class="adsbygoogle" style="display:block" data-ad-client="ca-pub-9333911690486629" data-ad-slot="2879047309" data-ad-format="auto" data-full-width-responsive="true"></ins> <script> (adsbygoogle = window.adsbygoogle || []).push({}); </script></div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/32452549/Lanadelumab-demonstrates-rapid-and-sustained-prevention-of-hereditary-angioedema-attacks" >Lanadelumab demonstrates rapid and sustained prevention of hereditary angioedema attacks.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> <a href='https://www.pubfacts.com/author/Marcus+Maurer'>Marcus Maurer</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> <a href='https://www.pubfacts.com/author/Hilary+J+Longhurst'>Hilary J Longhurst</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/Peng+Lu'>Peng Lu</a> <a href='https://www.pubfacts.com/author/James+Hao'>James Hao</a> <a href='https://www.pubfacts.com/author/Salom%C3%A9+Juethner'>Salomé Juethner</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/+'> </a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Allergy 2020 11 6;75(11):2879-2887. Epub 2020 Jul 6.</p><p>Allergy and Asthma Research Associates, Dallas, TX, USA.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: Lanadelumab demonstrated efficacy in preventing hereditary angioedema (HAE) attacks in the phase 3 HELP Study.<BR><BR><em>Objective</em>: To assess time to onset of effect and long-term efficacy of lanadelumab, based on exploratory findings from the HELP Study.<BR><BR><em>Methods</em>: Eligible patients with HAE type I/II received lanadelumab 150 mg every 4 weeks (q4wks), 300 mg q4wks, 300 mg q2wks, or placebo. Ad hoc analyses evaluated day 0-69 findings using a Poisson regression model accounting for overdispersion. Least-squares mean monthly HAE attack rate for lanadelumab was compared with placebo. Intrapatient comparisons for days 0-69 versus steady state (days 70-182) used a paired t test for continuous endpoints or Kappa statistics for categorical endpoints.<BR><BR><em>Results</em>: One hundred twenty-five patients were randomized and treated. During days 0-69, mean monthly attack rate was significantly lower with lanadelumab (0.41-0.76) vs placebo (2.04), including attacks requiring acute treatment (0.33-0.61 vs 1.66) and moderate/severe attacks (0.31-0.48 vs 1.33, all P ≤ .001). More patients receiving lanadelumab vs placebo were attack free (37.9%-48.1% vs 7.3%) and responders (85.7%-100% vs 26.8%). During steady state, the efficacy of lanadelumab vs placebo was similar or improved vs days 0-69. Intrapatient differences were significant with lanadelumab 300 mg q4wks for select outcomes. Lanadelumab efficacy was durable-HAE attack rate was consistently lower vs placebo, from the first 2 weeks of treatment through study end. Treatment emergent adverse events were comparable during days 0-69 and 70-182.<BR><BR><em>Conclusion</em>: Protection with lanadelumab started from the first dose and continued throughout the entire study period.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/32452549/Lanadelumab-demonstrates-rapid-and-sustained-prevention-of-hereditary-angioedema-attacks" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1111/all.14416' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-32452549-n' >http://dx.doi.org/10.1111/all.14416</a></td><td>DOI Listing</td></tr><tr><td><i class='icon-doc'></i><a href='http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7689768' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_PMC", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-32452549-n'>http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7689768</a></td><td>PMC</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> November 2020</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/32452549" target="_blank" title="" class="fb socialShare" id="fb-a-32452549"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/32452549" target="_blank" title="" class="tw socialShare" id="tw-a-32452549"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/32452549&title=Lanadelumab+demonstrates+rapid+and+sustained+prevention+of+hereditary+angioedema+attacks.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-32452549"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/31621458/Immune-globulin-subcutaneous-human-klhw-20-for-primary-humoral-immunodeficiency-an-open-label-Phase-" >Immune globulin subcutaneous, human - klhw 20% for primary humoral immunodeficiency: an open-label, Phase III study.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/John+W+Sleasman'>John W Sleasman</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Iftikhar+Hussain'>Iftikhar Hussain</a> <a href='https://www.pubfacts.com/author/H+James+Wedner'>H James Wedner</a> <a href='https://www.pubfacts.com/author/James+B+Harris'>James B Harris</a> <a href='https://www.pubfacts.com/author/Kecia+L+Courtney'>Kecia L Courtney</a> <a href='https://www.pubfacts.com/author/Elsa+Mondou'>Elsa Mondou</a> <a href='https://www.pubfacts.com/author/Jiang+Lin'>Jiang Lin</a> <a href='https://www.pubfacts.com/author/Mark+R+Stein'>Mark R Stein</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Immunotherapy 2019 11 17;11(16):1371-1386. Epub 2019 Oct 17.</p><p>Good Samaritan Medical Center; 1309 N Flagler Dr, West Palm Beach, FL 33401, USA.</p><div class="pubDescriptor" style="margin-top:20px;"> This prospective, Phase III study assessed the pharmacokinetics (PK), safety and tolerability of immune globulin subcutaneous, human - klhw 20% solution (IGSC-C 20%) in participants with primary humoral immunodeficiency (PI), compared with immune globulin injection (human), 10% caprylate/chromatography purified (IGIV-C 10%). About 53 participants enrolled. Total 44 received IGIV-C 10% in the run-in phase and then entered the IV phase (with an additional nine who were already receiving IGIV-C 10% and entered the IV phase directly) for steady-state IV PK assessments. Total 49 entered the SC phase (weekly doses of IGSC-C 20% for ∼24 weeks). The PK profiles of IGIV-C 10% and IGSC-C 20% and their safety and tolerability parameters were compared. At a dose adjustment factor of 1.37, IGSC-C 20% provided comparable (noninferior and bioequivalent) overall total immunoglobulin G exposure to IGIV-C 10% over an equal time interval. About 33 participants reported 79 adverse events during run-in + IV phases; 41 participants reported 141 adverse events during the SC phase, with most being local infusion site reactions. The majority of infusion site reactions were mild to moderate in severity. IGSC-C 20% was bioequivalent to IGIV-C 10% and was well tolerated, with a safety profile comparable with IGIV-C 10%, in this study. Trial registration: ClinicalTrials.gov identifier: NCT02604810.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/31621458/Immune-globulin-subcutaneous-human-klhw-20-for-primary-humoral-immunodeficiency-an-open-label-Phase-" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.2217/imt-2019-0159' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-31621458-n' >http://dx.doi.org/10.2217/imt-2019-0159</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> November 2019</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/31621458" target="_blank" title="" class="fb socialShare" id="fb-a-31621458"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/31621458" target="_blank" title="" class="tw socialShare" id="tw-a-31621458"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/31621458&title=Immune+globulin+subcutaneous%2C+human+-+klhw+20%25+for+primary+humoral+immunodeficiency%3A+an+open-label%2C+Phase+III+study.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-31621458"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/31170541/Safety-of-recombinant-human-C1-esterase-inhibitor-for-hereditary-angioedema-attacks-during-pregnancy" >Safety of recombinant human C1 esterase inhibitor for hereditary angioedema attacks during pregnancy.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Dumitru+Moldovan'>Dumitru Moldovan</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/Roman+Hakl'>Roman Hakl</a> <a href='https://www.pubfacts.com/author/Grzegorz+Porebski'>Grzegorz Porebski</a> <a href='https://www.pubfacts.com/author/Kimberly+Poarch'>Kimberly Poarch</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Anurag+Relan'>Anurag Relan</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>J Allergy Clin Immunol Pract 2019 Nov - Dec;7(8):2938-2940. Epub 2019 Jun 3.</p><p>Pharming Healthcare Inc, Bridgewater, NJ.</p><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/31170541/Safety-of-recombinant-human-C1-esterase-inhibitor-for-hereditary-angioedema-attacks-during-pregnancy" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.jaip.2019.05.042' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-31170541-n' >http://dx.doi.org/10.1016/j.jaip.2019.05.042</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> October 2020</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/31170541" target="_blank" title="" class="fb socialShare" id="fb-a-31170541"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/31170541" target="_blank" title="" class="tw socialShare" id="tw-a-31170541"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/31170541&title=Safety+of+recombinant+human+C1+esterase+inhibitor+for+hereditary+angioedema+attacks+during+pregnancy.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-31170541"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/30682573/Fixed-Dose-Subcutaneous-C1-Inhibitor-Liquid-for-Prophylactic-Treatment-of-C1-INH-HAE-SAHARA-Randomiz" >Fixed-Dose Subcutaneous C1-Inhibitor Liquid for Prophylactic Treatment of C1-INH-HAE: SAHARA Randomized Study.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Inmaculada+Martinez-Saguer'>Inmaculada Martinez-Saguer</a> <a href='https://www.pubfacts.com/author/William+H+Yang'>William H Yang</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/Joshua+Jacobs'>Joshua Jacobs</a> <a href='https://www.pubfacts.com/author/Dumitru+Moldovan'>Dumitru Moldovan</a> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> <a href='https://www.pubfacts.com/author/Douglas+T+Johnston'>Douglas T Johnston</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/Yongqiang+Tang'>Yongqiang Tang</a> <a href='https://www.pubfacts.com/author/Jennifer+Schranz'>Jennifer Schranz</a> <a href='https://www.pubfacts.com/author/Peng+Lu'>Peng Lu</a> <a href='https://www.pubfacts.com/author/Moshe+Vardi'>Moshe Vardi</a> <a href='https://www.pubfacts.com/author/Henriette+Farkas'>Henriette Farkas</a> <a href='https://www.pubfacts.com/author/+'> </a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>J Allergy Clin Immunol Pract 2019 May - Jun;7(5):1610-1618.e4. Epub 2019 Jan 23.</p><p>Hungarian Angioedema Reference Center, Semmelweis University, Budapest, Hungary.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: Hereditary angioedema (HAE) with C1 inhibitor deficiency (C1-INH) is characterized by swelling of subcutaneous and/or submucosal tissues.<BR><BR><em>Objective</em>: To evaluate efficacy/safety of fixed-dose subcutaneous plasma-derived C1-INH (pdC1-INH) liquid for HAE attack prevention (NCT02584959).<BR><BR><em>Methods</em>: Eligible patients were ≥12 years with ≥2 monthly attacks prescreening or pre-long-term prophylaxis. In a partial crossover design, 80% of patients were randomized to placebo or pdC1-INH liquid for 14 weeks and crossed over from active to placebo or vice versa for another 14 weeks. The remainder were randomized to pdC1-INH liquid for 28 weeks. The primary efficacy endpoint was normalized number of attacks (NNA) versus placebo. Key additional endpoints were the proportion of patients achieving NNA reduction ≥50%, attack severity, number of attack-free days, and safety.<BR><BR><em>Results</em>: Seventy-five patients were randomized and 58 (77%) completed the study. Mean age 41 years; 88% HAE type I. Least-squares means of NNA were reduced from 3.9 with placebo to 1.6 with pdC1-INH (from day 1; P < .0001). Most patients had ≥50% NNA reduction with pdC1-INH (from day 1, 78%). A total of 8.8% of placebo-treated patients were attack-free and 5.3%, 22.8%, and 63.2% had mild, moderate, and severe attacks, respectively; 37.5% of pdC1-INH-treated patients were attack-free and 8.9%, 26.8%, and 26.8% had mild, moderate, and severe attacks, respectively. Treatment-emergent adverse event rates were similar between groups (52% vs 56% for pdC1-INH crossover vs placebo, respectively).<BR><BR><em>Conclusions</em>: Fixed-dose subcutaneous pdC1-INH liquid was superior to placebo in preventing HAE attacks and demonstrated a favorable safety profile.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/30682573/Fixed-Dose-Subcutaneous-C1-Inhibitor-Liquid-for-Prophylactic-Treatment-of-C1-INH-HAE-SAHARA-Randomiz" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.jaip.2019.01.021' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-30682573-n' >http://dx.doi.org/10.1016/j.jaip.2019.01.021</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> August 2020</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/30682573" target="_blank" title="" class="fb socialShare" id="fb-a-30682573"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/30682573" target="_blank" title="" class="tw socialShare" id="tw-a-30682573"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/30682573&title=Fixed-Dose+Subcutaneous+C1-Inhibitor+Liquid+for+Prophylactic+Treatment+of+C1-INH-HAE%3A+SAHARA+Randomized+Study.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-30682573"><i class="icon-linkedin"></i></a> </div></div> </div> </div> <div class="row" > <div class="col-md-12" style="text-align:center;" ><script async src="https://pagead2.googlesyndication.com/pagead/js/adsbygoogle.js"></script> <!-- 2) Display - Horizontal - Author - Responsive --> <ins class="adsbygoogle" style="display:block" data-ad-client="ca-pub-9333911690486629" data-ad-slot="2879047309" data-ad-format="auto" data-full-width-responsive="true"></ins> <script> (adsbygoogle = window.adsbygoogle || []).push({}); </script></div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/30539362/Lanadelumab-for-the-Prophylactic-Treatment-of-Hereditary-Angioedema-with-C1-Inhibitor-Deficiency-A-R" >Lanadelumab for the Prophylactic Treatment of Hereditary Angioedema with C1 Inhibitor Deficiency: A Review of Preclinical and Phase I Studies.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Paula+J+Busse'>Paula J Busse</a> <a href='https://www.pubfacts.com/author/Henriette+Farkas'>Henriette Farkas</a> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Hilary+J+Longhurst'>Hilary J Longhurst</a> <a href='https://www.pubfacts.com/author/Daniel+J+Sexton'>Daniel J Sexton</a> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>BioDrugs 2019 Feb;33(1):33-43</p><p>Division of Rheumatology, Allergy and Immunology, University of California, San Diego, 8899 University Center Lane, Suite 230, San Diego, CA, 92122, USA.</p><div class="pubDescriptor" style="margin-top:20px;">Hereditary angioedema (HAE) with C1 esterase inhibitor (C1-INH) deficiency (C1-INH-HAE) is a rare disease characterized by diminished levels or dysfunctional activity of C1-INH, leading to dysregulated plasma kallikrein activity within the kallikrein-kinin pathway. Symptoms manifest as painful, potentially life-threatening swelling of subcutaneous tissues throughout the body and/or submucosal edema in the upper airway or gastrointestinal tract. Attacks recur with unpredictable frequency, intensity, and duration, placing a heavy burden on patients' daily lives. Despite improved availability of medications for on-demand treatment during attacks and prophylaxis of future attacks, unmet needs remain. Lanadelumab, a fully human monoclonal antibody, may help address some of the limitations of existing prophylactic options (e.g., the need for intravenous administration or frequent dosing). Preclinical studies demonstrate that it is highly potent and specifically inhibits plasma kallikrein, and findings from phase Ia and Ib studies suggest this agent is well tolerated and provides sustained inhibition of plasma kallikrein, allowing for less frequent dosing. The phase III HELP Study (NCT02586805) evaluating the efficacy and safety of lanadelumab in preventing HAE attacks has been completed, and its open-label extension (NCT02741596) is ongoing. Lanadelumab is now approved in the USA and Canada for prophylaxis to prevent attacks of HAE in patients aged ≥ 12 years. This review provides an overview of the discovery and clinical development of lanadelumab, from preclinical through phase Ib studies, characterizing its safety/tolerability, efficacy, and pharmacokinetic and pharmacodynamic profiles. It also highlights how this agent may positively impact clinical care of patients with C1-INH-HAE.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/30539362/Lanadelumab-for-the-Prophylactic-Treatment-of-Hereditary-Angioedema-with-C1-Inhibitor-Deficiency-A-R" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1007/s40259-018-0325-y' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-30539362-n' >http://dx.doi.org/10.1007/s40259-018-0325-y</a></td><td>DOI Listing</td></tr><tr><td><i class='icon-doc'></i><a href='http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6373397' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_PMC", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-30539362-n'>http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6373397</a></td><td>PMC</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> February 2019</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/30539362" target="_blank" title="" class="fb socialShare" id="fb-a-30539362"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/30539362" target="_blank" title="" class="tw socialShare" id="tw-a-30539362"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/30539362&title=Lanadelumab+for+the+Prophylactic+Treatment+of+Hereditary+Angioedema+with+C1+Inhibitor+Deficiency%3A+A+Review+of+Preclinical+and+Phase+I+Studies.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-30539362"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/30480729/Effect-of-Lanadelumab-Compared-With-Placebo-on-Prevention-of-Hereditary-Angioedema-Attacks-A-Randomi" >Effect of Lanadelumab Compared With Placebo on Prevention of Hereditary Angioedema Attacks: A Randomized Clinical Trial.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/Marco+Cicardi'>Marco Cicardi</a> <a href='https://www.pubfacts.com/author/Hilary+J+Longhurst'>Hilary J Longhurst</a> <a href='https://www.pubfacts.com/author/Bruce+L+Zuraw'>Bruce L Zuraw</a> <a href='https://www.pubfacts.com/author/Paula+J+Busse'>Paula J Busse</a> <a href='https://www.pubfacts.com/author/John+Anderson'>John Anderson</a> <a href='https://www.pubfacts.com/author/Markus+Magerl'>Markus Magerl</a> <a href='https://www.pubfacts.com/author/Inmaculada+Martinez-Saguer'>Inmaculada Martinez-Saguer</a> <a href='https://www.pubfacts.com/author/Mark+Davis-Lorton'>Mark Davis-Lorton</a> <a href='https://www.pubfacts.com/author/Andrea+Zanichelli'>Andrea Zanichelli</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/Timothy+Craig'>Timothy Craig</a> <a href='https://www.pubfacts.com/author/Joshua+Jacobs'>Joshua Jacobs</a> <a href='https://www.pubfacts.com/author/Douglas+T+Johnston'>Douglas T Johnston</a> <a href='https://www.pubfacts.com/author/Ralph+Shapiro'>Ralph Shapiro</a> <a href='https://www.pubfacts.com/author/William+H+Yang'>William H Yang</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Michael+E+Manning'>Michael E Manning</a> <a href='https://www.pubfacts.com/author/Lawrence+B+Schwartz'>Lawrence B Schwartz</a> <a href='https://www.pubfacts.com/author/Mustafa+Shennak'>Mustafa Shennak</a> <a href='https://www.pubfacts.com/author/Daniel+Soteres'>Daniel Soteres</a> <a href='https://www.pubfacts.com/author/Rafael+H+Zaragoza-Urdaz'>Rafael H Zaragoza-Urdaz</a> <a href='https://www.pubfacts.com/author/Selina+Gierer'>Selina Gierer</a> <a href='https://www.pubfacts.com/author/Andrew+M+Smith'>Andrew M Smith</a> <a href='https://www.pubfacts.com/author/Raffi+Tachdjian'>Raffi Tachdjian</a> <a href='https://www.pubfacts.com/author/H+James+Wedner'>H James Wedner</a> <a href='https://www.pubfacts.com/author/Jacques+Hebert'>Jacques Hebert</a> <a href='https://www.pubfacts.com/author/Syed+M+Rehman'>Syed M Rehman</a> <a href='https://www.pubfacts.com/author/Petra+Staubach'>Petra Staubach</a> <a href='https://www.pubfacts.com/author/Jennifer+Schranz'>Jennifer Schranz</a> <a href='https://www.pubfacts.com/author/Jovanna+Baptista'>Jovanna Baptista</a> <a href='https://www.pubfacts.com/author/Wolfram+Nothaft'>Wolfram Nothaft</a> <a href='https://www.pubfacts.com/author/Marcus+Maurer'>Marcus Maurer</a> <a href='https://www.pubfacts.com/author/+'> </a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>JAMA 2018 11;320(20):2108-2121</p><p>Department of Dermatology and Allergy, Dermatological Allergology, Charité-Universitätsmedizin Berlin, Berlin, Germany.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Importance</em>: Current treatments for long-term prophylaxis in hereditary angioedema have limitations.<BR><BR><em>Objective</em>: To assess the efficacy of lanadelumab, a fully human monoclonal antibody that selectively inhibits active plasma kallikrein, in preventing hereditary angioedema attacks.<BR><BR><em>Design, Setting, And Participants</em>: Phase 3, randomized, double-blind, parallel-group, placebo-controlled trial conducted at 41 sites in Canada, Europe, Jordan, and the United States. Patients were randomized between March 3, 2016, and September 9, 2016; last day of follow-up was April 13, 2017. Randomization was 2:1 lanadelumab to placebo; patients assigned to lanadelumab were further randomized 1:1:1 to 1 of the 3 dose regimens. Patients 12 years or older with hereditary angioedema type I or II underwent a 4-week run-in period and those with 1 or more hereditary angioedema attacks during run-in were randomized.<BR><BR><em>Interventions</em>: Twenty-six-week treatment with subcutaneous lanadelumab 150 mg every 4 weeks (n = 28), 300 mg every 4 weeks (n = 29), 300 mg every 2 weeks (n = 27), or placebo (n = 41). All patients received injections every 2 weeks, with those in the every-4-week group receiving placebo in between active treatments.<BR><BR><em>Main Outcome And Measures</em>: Primary efficacy end point was the number of investigator-confirmed attacks of hereditary angioedema over the treatment period.<BR><BR><em>Results</em>: Among 125 patients randomized (mean age, 40.7 years [SD, 14.7 years]; 88 females [70.4%]; 113 white [90.4%]), 113 (90.4%) completed the study. During the run-in period, the mean number of hereditary angioedema attacks per month in the placebo group was 4.0; for the lanadelumab groups, 3.2 for the every-4-week 150-mg group; 3.7 for the every-4-week 300-mg group; and 3.5 for the every-2-week 300-mg group. During the treatment period, the mean number of attacks per month for the placebo group was 1.97; for the lanadelumab groups, 0.48 for the every-4-week 150-mg group; 0.53 for the every-4-week 300-mg group; and 0.26 for the every-2-week 300-mg group. Compared with placebo, the mean differences in the attack rate per month were -1.49 (95% CI, -1.90 to -1.08; P < .001); -1.44 (95% CI, -1.84 to -1.04; P < .001); and -1.71 (95% CI, -2.09 to -1.33; P < .001). The most commonly occurring adverse events with greater frequency in the lanadelumab treatment groups were injection site reactions (34.1% placebo, 52.4% lanadelumab) and dizziness (0% placebo, 6.0% lanadelumab).<BR><BR><em>Conclusions And Relevance</em>: Among patients with hereditary angioedema type I or II, treatment with subcutaneous lanadelumab for 26 weeks significantly reduced the attack rate compared with placebo. These findings support the use of lanadelumab as a prophylactic therapy for hereditary angioedema. Further research is needed to determine long-term safety and efficacy.<BR><BR><em>Trial Registration</em>: EudraCT Identifier: 2015-003943-20; ClinicalTrials.gov Identifier: NCT02586805.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/30480729/Effect-of-Lanadelumab-Compared-With-Placebo-on-Prevention-of-Hereditary-Angioedema-Attacks-A-Randomi" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1001/jama.2018.16773' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-30480729-n' >http://dx.doi.org/10.1001/jama.2018.16773</a></td><td>DOI Listing</td></tr><tr><td><i class='icon-doc'></i><a href='http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6583584' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_PMC", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-30480729-n'>http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6583584</a></td><td>PMC</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> November 2018</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/30480729" target="_blank" title="" class="fb socialShare" id="fb-a-30480729"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/30480729" target="_blank" title="" class="tw socialShare" id="tw-a-30480729"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/30480729&title=Effect+of+Lanadelumab+Compared+With+Placebo+on+Prevention+of+Hereditary+Angioedema+Attacks%3A+A+Randomized+Clinical+Trial.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-30480729"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/30132644/Current-and-emerging-therapies-to-prevent-hereditary-angioedema-attacks" >Current and emerging therapies to prevent hereditary angioedema attacks.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Am J Manag Care 2018 08;24(14 Suppl):S299-S307</p><p>Clinical Professor of Internal Medicine, Allergy/Immunology Division, University of Texas Southwestern Medical School, private practice, Dallas, TX. Email: </p><div class="pubDescriptor" style="margin-top:20px;">Hereditary angioedema (HAE) is a rare genetic disease defined by recurrent attacks of edema, causing a substantial burden for patients, with morbidity, mortality, and reduced quality of life. This burden is increased by delayed diagnosis, inappropriate treatment, and suboptimal follow-up and patient education. Several novel therapeutics have recently been approved or are currently under evaluation for prevention of HAE attacks.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/30132644/Current-and-emerging-therapies-to-prevent-hereditary-angioedema-attacks" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> August 2018</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/30132644" target="_blank" title="" class="fb socialShare" id="fb-a-30132644"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/30132644" target="_blank" title="" class="tw socialShare" id="tw-a-30132644"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/30132644&title=Current+and+emerging+therapies+to+prevent+hereditary+angioedema+attacks.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-30132644"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/30056152/Long-term-prophylaxis-therapy-in-patients-with-hereditary-angioedema-with-C1-inhibitor-deficiency" >Long-term prophylaxis therapy in patients with hereditary angioedema with C1 inhibitor deficiency.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Timothy+Craig'>Timothy Craig</a> <a href='https://www.pubfacts.com/author/Paula+Busse'>Paula Busse</a> <a href='https://www.pubfacts.com/author/Richard+G+Gower'>Richard G Gower</a> <a href='https://www.pubfacts.com/author/Douglas+T+Johnston'>Douglas T Johnston</a> <a href='https://www.pubfacts.com/author/Jay+M+Kashkin'>Jay M Kashkin</a> <a href='https://www.pubfacts.com/author/Huamin+H+Li'>Huamin H Li</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> <a href='https://www.pubfacts.com/author/Daniel+Soteres'>Daniel Soteres</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Ann Allergy Asthma Immunol 2018 12 27;121(6):673-679. Epub 2018 Jul 27.</p><p>Asthma and Allergy Associates PC, Colorado Springs, Colorado.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Objective</em>: To review the criteria for long-term prophylaxis therapy in patients with hereditary angioedema due to C1 inhibitor deficiency (C1-INH-HAE), describe how these criteria have evolved over time, and anticipate how criteria may change in the future with the availability of new C1-INH-HAE treatment options.<BR><BR><em>Data Sources</em>: Treatment guidelines, consensus statements, and expert reviews.<BR><BR><em>Study Selections</em>: Manuscripts that described long-term prophylaxis therapy in patients with C1-INH-HAE were selected.<BR><BR><em>Results</em>: Historically, patients with C1-INH-HAE were considered to be candidates for long-term prophylaxis therapy if they had at least 1 attack per month, had at least 5 days of disability per month because of C1-INH-HAE, or did not sufficiently respond to on-demand treatment. More recently, guidelines and reviews state that thresholds of number of attacks or days of disability are arbitrary and that treatment plans should be individualized to the patient's needs. Furthermore, all patients should have a comprehensive management plan that is reviewed periodically and should have at least 2 doses of on-demand treatment available. Prophylaxis therapy should be discussed as a potential treatment option for each patient; however, the decision for its use will depend on the patient's individual needs and the course of their symptoms.<BR><BR><em>Conclusion</em>: The criteria for long-term prophylaxis therapy in C1-INH-HAE have changed with the recognition that treatments should be individualized to the patient's needs and with the availability of new medications that have more favorable benefit-risk profiles, are easier to use, and improve patients' quality of life.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/30056152/Long-term-prophylaxis-therapy-in-patients-with-hereditary-angioedema-with-C1-inhibitor-deficiency" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.anai.2018.07.025' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-30056152-n' >http://dx.doi.org/10.1016/j.anai.2018.07.025</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> December 2018</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/30056152" target="_blank" title="" class="fb socialShare" id="fb-a-30056152"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/30056152" target="_blank" title="" class="tw socialShare" id="tw-a-30056152"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/30056152&title=Long-term+prophylaxis+therapy+in+patients+with+hereditary+angioedema+with+C1+inhibitor+deficiency.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-30056152"><i class="icon-linkedin"></i></a> </div></div> </div> </div> <div class="row" > <div class="col-md-12" style="text-align:center;" ><script async src="https://pagead2.googlesyndication.com/pagead/js/adsbygoogle.js"></script> <!-- 2) Display - Horizontal - Author - Responsive --> <ins class="adsbygoogle" style="display:block" data-ad-client="ca-pub-9333911690486629" data-ad-slot="2879047309" data-ad-format="auto" data-full-width-responsive="true"></ins> <script> (adsbygoogle = window.adsbygoogle || []).push({}); </script></div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/29998524/Population-pharmacokinetics-of-subcutaneous-C1-inhibitor-for-prevention-of-attacks-in-patients-with-" >Population pharmacokinetics of subcutaneous C1-inhibitor for prevention of attacks in patients with hereditary angioedema.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Dipti+Pawaskar'>Dipti Pawaskar</a> <a href='https://www.pubfacts.com/author/Michael+A+Tortorici'>Michael A Tortorici</a> <a href='https://www.pubfacts.com/author/Bruce+Zuraw'>Bruce Zuraw</a> <a href='https://www.pubfacts.com/author/Timothy+Craig'>Timothy Craig</a> <a href='https://www.pubfacts.com/author/Marco+Cicardi'>Marco Cicardi</a> <a href='https://www.pubfacts.com/author/Hilary+Longhurst'>Hilary Longhurst</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Inmaculada+Martinez-Saguer'>Inmaculada Martinez-Saguer</a> <a href='https://www.pubfacts.com/author/Joshua+Jacobs'>Joshua Jacobs</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> <a href='https://www.pubfacts.com/author/Constance+H+Katelaris'>Constance H Katelaris</a> <a href='https://www.pubfacts.com/author/Paul+K+Keith'>Paul K Keith</a> <a href='https://www.pubfacts.com/author/Annette+Feussner'>Annette Feussner</a> <a href='https://www.pubfacts.com/author/Jagdev+Sidhu'>Jagdev Sidhu</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Clin Exp Allergy 2018 10 26;48(10):1325-1332. Epub 2018 Aug 26.</p><p>CSL Limited, Parkville, Melbourne, Australia.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: Long-term prophylaxis with subcutaneous (SC) administration of a highly concentrated plasma-derived C1-esterase inhibitor (C1-INH) formulation was recently approved by the Food and Drug Administration for hereditary angioedema (HAE) attack prevention.<BR><BR><em>Objective</em>: To characterize the population pharmacokinetics of C1-INH (SC) (HAEGARDA ; CSL Behring) in healthy volunteers and HAE patients, and assess the variability and influence of covariates on pharmacokinetics.<BR><BR><em>Methods</em>: C1-INH functional activity data obtained after administration of various C1-INH (intravenous; IV) and C1-INH (SC) doses from 1 study in healthy volunteers (n = 16) and 2 studies in subjects with HAE (n = 108) were pooled to develop a population pharmacokinetic model (NONMEM v7.2). Pharmacokinetic parameters derived from steady-state simulations based on the final model were also evaluated.<BR><BR><em>Results</em>: C1-INH functional activity following C1-INH (SC) administration was described by a linear one-compartment model with first-order absorption and elimination, with inter-individual variability in all parameters tested. The mean population bioavailability of C1-INH (SC), and pharmacokinetic parameters for clearance (CL), volume of distribution, and absorption rate were estimated to be ~43%, 1.03 mL/hour/kg, 0.05 L/kg and 0.0146 hour , respectively. The effect of bodyweight on CL of C1-INH functional activity was included in the final model, estimated to be 0.74. Steady-state simulations of C1-INH functional activity vs time profiles in 1000 virtual HAE patients revealed higher minimum functional activity (C ) levels after twice-weekly dosing with 40 IU/kg (~40%) and 60 IU/kg (~48%) compared with 1000 IU IV (~30%). Based on the population pharmacokinetic model, the median time to peak concentration was ~59 hours and the median apparent plasma half-life was ~69 hours.<BR><BR><em>Conclusions And Clinical Relevance</em>: Twice-weekly bodyweight-adjusted dosing of C1-INH (SC) exhibits linear pharmacokinetics and dose-dependent increases in C levels at each dosing interval. In this analysis, SC dosing led to maintenance of higher C levels than IV dosing.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/29998524/Population-pharmacokinetics-of-subcutaneous-C1-inhibitor-for-prevention-of-attacks-in-patients-with-" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1111/cea.13220' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-29998524-n' >http://dx.doi.org/10.1111/cea.13220</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> October 2018</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/29998524" target="_blank" title="" class="fb socialShare" id="fb-a-29998524"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/29998524" target="_blank" title="" class="tw socialShare" id="tw-a-29998524"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/29998524&title=Population+pharmacokinetics+of+subcutaneous+C1-inhibitor+for+prevention+of+attacks+in+patients+with+hereditary+angioedema.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-29998524"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/29391286/Health-Related-Quality-of-Life-with-Subcutaneous-C1-Inhibitor-for-Prevention-of-Attacks-of-Hereditar" >Health-Related Quality of Life with Subcutaneous C1-Inhibitor for Prevention of Attacks of Hereditary Angioedema.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Timothy+Craig'>Timothy Craig</a> <a href='https://www.pubfacts.com/author/Bruce+Zuraw'>Bruce Zuraw</a> <a href='https://www.pubfacts.com/author/Hilary+Longhurst'>Hilary Longhurst</a> <a href='https://www.pubfacts.com/author/James+Baker'>James Baker</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/John+Anderson'>John Anderson</a> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> <a href='https://www.pubfacts.com/author/Michael+E+Manning'>Michael E Manning</a> <a href='https://www.pubfacts.com/author/Paul+K+Keith'>Paul K Keith</a> <a href='https://www.pubfacts.com/author/Donald+S+Levy'>Donald S Levy</a> <a href='https://www.pubfacts.com/author/Teresa+Caballero'>Teresa Caballero</a> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> <a href='https://www.pubfacts.com/author/Richard+G+Gower'>Richard G Gower</a> <a href='https://www.pubfacts.com/author/Henriette+Farkas'>Henriette Farkas</a> <a href='https://www.pubfacts.com/author/John-Philip+Lawo'>John-Philip Lawo</a> <a href='https://www.pubfacts.com/author/Ingo+Pragst'>Ingo Pragst</a> <a href='https://www.pubfacts.com/author/Thomas+Machnig'>Thomas Machnig</a> <a href='https://www.pubfacts.com/author/Douglas+J+Watson'>Douglas J Watson</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>J Allergy Clin Immunol Pract 2018 Sep - Oct;6(5):1733-1741.e3. Epub 2018 Jan 31.</p><p>CSL Behring, King of Prussia, Pa.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: Hereditary angioedema with C1-inhibitor deficiency (C1-INH-HAE) impairs health-related quality of life (HRQoL).<BR><BR><em>Objective</em>: The objective of this study was to assess HRQoL outcomes in patients self-administering subcutaneous C1-INH (C1-INH[SC]; HAEGARDA) for routine prevention of HAE attacks.<BR><BR><em>Methods</em>: Post hoc analysis of data from the placebo-controlled, crossover phase III COMPACT study (Clinical Studies for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1-Inhibitor Replacement Therapy). Ninety patients with C1-INH-HAE were randomized to 1 of 4 treatment sequences: C1-INH(SC) 40 or 60 IU/kg twice weekly for 16 weeks, preceded or followed by 16 weeks of twice weekly placebo injections. All HAE attacks were treated with open-label on-demand treatment as necessary. HRQoL assessments at week 14 (last visit) included the European Quality of Life-5 Dimensions Questionnaire (EQ-5D-3L), the Hospital Anxiety and Depression Scale (HADS), the Work Productivity and Activity Impairment Questionnaire (WPAI), and the Treatment Satisfaction Questionnaire for Medication (TSQM).<BR><BR><em>Results</em>: Compared with placebo (on-demand treatment alone), treatment with twice weekly C1-INH(SC) (both doses combined) was associated with better EQ-5D visual analog scale general health, less HADS anxiety, less WPAI presenteeism, work productivity loss, and activity impairment, and greater TSQM effectiveness and overall treatment satisfaction. More patients self-reported a "good/excellent" response during routine prevention with C1-INH(SC) compared with on-demand only (placebo prophylaxis) management. For each HRQoL measure, a greater proportion of patients had a clinically meaningful improvement during C1-INH(SC) treatment compared with placebo.<BR><BR><em>Conclusions</em>: In patients with frequent HAE attacks, a treatment strategy of routine prevention with self-administered twice weekly C1-INH(SC) had a greater impact on improving multiple HAE-related HRQoL impairments, most notably anxiety and work productivity, compared with on-demand treatment alone (placebo prophylaxis).</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/29391286/Health-Related-Quality-of-Life-with-Subcutaneous-C1-Inhibitor-for-Prevention-of-Attacks-of-Hereditar" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.jaip.2017.12.039' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-29391286-n' >http://dx.doi.org/10.1016/j.jaip.2017.12.039</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> November 2019</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/29391286" target="_blank" title="" class="fb socialShare" id="fb-a-29391286"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/29391286" target="_blank" title="" class="tw socialShare" id="tw-a-29391286"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/29391286&title=Health-Related+Quality+of+Life+with+Subcutaneous+C1-Inhibitor+for+Prevention+of+Attacks+of+Hereditary+Angioedema.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-29391286"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/28687113/Pharmacoeconomics-of-Orphan-Disease-Treatment-with-a-Focus-on-Hereditary-Angioedema" >Pharmacoeconomics of Orphan Disease Treatment with a Focus on Hereditary Angioedema.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Immunol Allergy Clin North Am 2017 08 13;37(3):617-628. Epub 2017 May 13.</p><p>Internal Medicine/Allergy/Immunology Division, University of Texas Southwestern Medical School, Dallas, TX, USA; AARA Research Associates, Private Practice, 10100 North Central Expressway, Suite 100, Dallas, TX 75231, USA. Electronic address: </p><div class="pubDescriptor" style="margin-top:20px;">This article discusses orphan diseases, their prevalence, legislative incentives to encourage development of therapies, and the impact of treatment on health care payment systems. Specifically, the cost burden of hereditary angioedema on patients, health care systems, and society is reviewed. The impact of availability of and access to novel and specific therapies on morbidity, mortality, and overall burden of disease is explored. Changes in treatment paradigms to improve effect and reduce cost of treatment are presented.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/28687113/Pharmacoeconomics-of-Orphan-Disease-Treatment-with-a-Focus-on-Hereditary-Angioedema" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.iac.2017.03.004' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-28687113-n' >http://dx.doi.org/10.1016/j.iac.2017.03.004</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> August 2017</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/28687113" target="_blank" title="" class="fb socialShare" id="fb-a-28687113"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/28687113" target="_blank" title="" class="tw socialShare" id="tw-a-28687113"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/28687113&title=Pharmacoeconomics+of+Orphan+Disease+Treatment+with+a+Focus+on+Hereditary+Angioedema.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-28687113"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/28328347/Prevention-of-Hereditary-Angioedema-Attacks-with-a-Subcutaneous-C1-Inhibitor" >Prevention of Hereditary Angioedema Attacks with a Subcutaneous C1 Inhibitor.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Hilary+Longhurst'>Hilary Longhurst</a> <a href='https://www.pubfacts.com/author/Marco+Cicardi'>Marco Cicardi</a> <a href='https://www.pubfacts.com/author/Timothy+Craig'>Timothy Craig</a> <a href='https://www.pubfacts.com/author/Konrad+Bork'>Konrad Bork</a> <a href='https://www.pubfacts.com/author/Clive+Grattan'>Clive Grattan</a> <a href='https://www.pubfacts.com/author/James+Baker'>James Baker</a> <a href='https://www.pubfacts.com/author/Huamin+H+Li'>Huamin H Li</a> <a href='https://www.pubfacts.com/author/Avner+Reshef'>Avner Reshef</a> <a href='https://www.pubfacts.com/author/James+Bonner'>James Bonner</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/John+Anderson'>John Anderson</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Henriette+Farkas'>Henriette Farkas</a> <a href='https://www.pubfacts.com/author/Constance+H+Katelaris'>Constance H Katelaris</a> <a href='https://www.pubfacts.com/author/Gordon+L+Sussman'>Gordon L Sussman</a> <a href='https://www.pubfacts.com/author/Joshua+Jacobs'>Joshua Jacobs</a> <a href='https://www.pubfacts.com/author/Marc+Riedl'>Marc Riedl</a> <a href='https://www.pubfacts.com/author/Michael+E+Manning'>Michael E Manning</a> <a href='https://www.pubfacts.com/author/Jacques+Hebert'>Jacques Hebert</a> <a href='https://www.pubfacts.com/author/Paul+K+Keith'>Paul K Keith</a> <a href='https://www.pubfacts.com/author/Shmuel+Kivity'>Shmuel Kivity</a> <a href='https://www.pubfacts.com/author/Sergio+Neri'>Sergio Neri</a> <a href='https://www.pubfacts.com/author/Donald+S+Levy'>Donald S Levy</a> <a href='https://www.pubfacts.com/author/Maria+L+Baeza'>Maria L Baeza</a> <a href='https://www.pubfacts.com/author/Robert+Nathan'>Robert Nathan</a> <a href='https://www.pubfacts.com/author/Lawrence+B+Schwartz'>Lawrence B Schwartz</a> <a href='https://www.pubfacts.com/author/Teresa+Caballero'>Teresa Caballero</a> <a href='https://www.pubfacts.com/author/William+Yang'>William Yang</a> <a href='https://www.pubfacts.com/author/Ioana+Crisan'>Ioana Crisan</a> <a href='https://www.pubfacts.com/author/Mar%C3%ADa+D+Hernandez'>María D Hernandez</a> <a href='https://www.pubfacts.com/author/Iftikhar+Hussain'>Iftikhar Hussain</a> <a href='https://www.pubfacts.com/author/Michael+Tarzi'>Michael Tarzi</a> <a href='https://www.pubfacts.com/author/Bruce+Ritchie'>Bruce Ritchie</a> <a href='https://www.pubfacts.com/author/Pavlina+Králíčková'>Pavlina Králíčková</a> <a href='https://www.pubfacts.com/author/Mar+Guilarte'>Mar Guilarte</a> <a href='https://www.pubfacts.com/author/Syed+M+Rehman'>Syed M Rehman</a> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> <a href='https://www.pubfacts.com/author/Richard+G+Gower'>Richard G Gower</a> <a href='https://www.pubfacts.com/author/Debra+Bensen-Kennedy'>Debra Bensen-Kennedy</a> <a href='https://www.pubfacts.com/author/Jonathan+Edelman'>Jonathan Edelman</a> <a href='https://www.pubfacts.com/author/Henrike+Feuersenger'>Henrike Feuersenger</a> <a href='https://www.pubfacts.com/author/John-Philip+Lawo'>John-Philip Lawo</a> <a href='https://www.pubfacts.com/author/Thomas+Machnig'>Thomas Machnig</a> <a href='https://www.pubfacts.com/author/Dipti+Pawaskar'>Dipti Pawaskar</a> <a href='https://www.pubfacts.com/author/Ingo+Pragst'>Ingo Pragst</a> <a href='https://www.pubfacts.com/author/Bruce+L+Zuraw'>Bruce L Zuraw</a> <a href='https://www.pubfacts.com/author/+'> </a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>N Engl J Med 2017 03;376(12):1131-1140</p><p>From Barts Health NHS Trust (H.L.) and St. John's Institute of Dermatology, Guy's Hospital (C.G.), London, and the Clinical Investigation and Research Unit, Royal Sussex County Hospital, Brighton (M.T.) - all in the United Kingdom; Ospedale Luigi Sacco-U.O. Medicina Generale, Milan (M.C.), and the Department of Internal Medicine, University of Catania, Catania (S.N.) - both in Italy; Department of Medicine and Pediatrics, Penn State Hershey Allergy, Asthma, and Immunology, Hershey (T. Craig), and CSL Behring, King of Prussia (D.B.-K., J.E., D.P.) - both in Pennsylvania; the Department of Dermatology, Johannes Gutenberg University Mainz, Mainz (K.B.), and CSL Behring, Marburg (H. Feuersenger, J.-P.L., T.M., I.P.) - both in Germany; Baker Allergy, Asthma and Dermatology Research Center, Portland, OR (J. Baker); Institute for Asthma and Allergy, Chevy Chase, MD (H.H.L.); Allergy and Immunology Unit, Chaim Sheba Medical Center, Tel Hashomer (A.R.), and Allergy and Immunology Unit, Tel Aviv Sourasky Medical Center, Tel Aviv (S.K.) - both in Israel; Clinical Research Center of Alabama, Birmingham (J. Bonner, J.A.); Department of Internal Medicine, Allergy Section Cincinnati, University of Cincinnati College of Medicine, Cincinnati (J.A.B.), and Toledo Institute of Clinical Research, Toledo (S.M.R.) - both in Ohio; Allergy Asthma Research Associates Research Center, Dallas (W.R.L.); Hungarian Angioedema Center, Third Department of Internal Medicine, Semmelweis University, Budapest (H. Farkas); the Department of Medicine, Immunology, and Allergy, Campbelltown Hospital, Campbelltown, NSW, Australia (C.H.K.); the Department of Clinical Immunology and Allergy, St. Michael's Hospital, Toronto (G.L.S.), Centre de Recherche Appliqué en Allergie de Québec, Quebec, QC (J.H.), McMaster University, Hamilton, ON (P.K.K.), Ottawa Allergy Research and University of Ottawa Medical School, Ottawa (W.Y.), and University of Alberta Hospital, Edmonton (B.R.) - all in Canada; Allergy and Asthma Clinical Research, Walnut Creek (J.J.), University of California, San Diego School of Medicine, La Jolla (M.R., B.L.Z.), and 705 W. La Veta Ave., Suite 101, Orange (D.S.L.) - all in California; Medical Research of Arizona, Scottsdale (M.E.M.); Hospital General Universitario Gregorio Marañón and Biomedical Research Network on Rare Diseases-U761, Institute for Health Research, Gregorio Marañón (M.L.B.), and the Allergy Department, Hospital La Paz Institute for Health Research, Biomedical Research Network on Rare Diseases (T. Caballero), Madrid, the Allergy Department, IIS Hospital Universitario La Fe, Valencia (M.D.H.), and Hospital Universitario Vall d'Hebron, Barcelona (M.G.) - all in Spain; Asthma and Allergy Association, Colorado Springs, CO (R.N.); Department of Internal Medicine, Virginia Commonwealth University, Richmond (L.B.S.); Spitalul Clinic Municipal, Cluj-Napoca, Romania (I.C.); Vital Prospects Clinical Research Institute, Tulsa, OK (I.H.); Institute of Clinical Immunology and Allergology, University Hospital, Hradec Kralove, Czech Republic (P.K.); Division of Rheumatology, Allergy, and Immunology, Massachusetts General Hospital, Boston (A.B.); and Marycliff Allergy Specialists, Spokane, WA (R.G.G.).</p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: Hereditary angioedema is a disabling, potentially fatal condition caused by deficiency (type I) or dysfunction (type II) of the C1 inhibitor protein. In a phase 2 trial, the use of CSL830, a nanofiltered C1 inhibitor preparation that is suitable for subcutaneous injection, resulted in functional levels of C1 inhibitor activity that would be expected to provide effective prophylaxis of attacks.<BR><BR><em>Methods</em>: We conducted an international, prospective, multicenter, randomized, double-blind, placebo-controlled, dose-ranging, phase 3 trial to evaluate the efficacy and safety of self-administered subcutaneous CSL830 in patients with type I or type II hereditary angioedema who had had four or more attacks in a consecutive 2-month period within 3 months before screening. We randomly assigned the patients to one of four treatment sequences in a crossover design, each involving two 16-week treatment periods: either 40 IU or 60 IU of CSL830 per kilogram of body weight twice weekly followed by placebo, or vice versa. The primary efficacy end point was the number of attacks of angioedema. Secondary efficacy end points were the proportion of patients who had a response (≥50% reduction in the number of attacks with CSL830 as compared with placebo) and the number of times that rescue medication was used.<BR><BR><em>Results</em>: Of the 90 patients who underwent randomization, 79 completed the trial. Both doses of CSL830, as compared with placebo, reduced the rate of attacks of hereditary angioedema (mean difference with 40 IU, -2.42 attacks per month; 95% confidence interval [CI], -3.38 to -1.46; and mean difference with 60 IU, -3.51 attacks per month; 95% CI, -4.21 to -2.81; P<0.001 for both comparisons). Response rates were 76% (95% CI, 62 to 87) in the 40-IU group and 90% (95% CI, 77 to 96) in the 60-IU group. The need for rescue medication was reduced from 5.55 uses per month in the placebo group to 1.13 uses per month in the 40-IU group and from 3.89 uses in the placebo group to 0.32 uses per month in the 60-IU group. Adverse events (most commonly mild and transient local site reactions) occurred in similar proportions of patients who received CSL830 and those who received placebo.<BR><BR><em>Conclusions</em>: In patients with hereditary angioedema, the prophylactic use of a subcutaneous C1 inhibitor twice weekly significantly reduced the frequency of acute attacks. (Funded by CSL Behring; COMPACT EudraCT number, 2013-000916-10 , and ClinicalTrials.gov number, NCT01912456 .).</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/28328347/Prevention-of-Hereditary-Angioedema-Attacks-with-a-Subcutaneous-C1-Inhibitor" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1056/NEJMoa1613627' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-28328347-n' >http://dx.doi.org/10.1056/NEJMoa1613627</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> March 2017</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/28328347" target="_blank" title="" class="fb socialShare" id="fb-a-28328347"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/28328347" target="_blank" title="" class="tw socialShare" id="tw-a-28328347"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/28328347&title=Prevention+of+Hereditary+Angioedema+Attacks+with+a+Subcutaneous+C1+Inhibitor.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-28328347"><i class="icon-linkedin"></i></a> </div></div> </div> </div> <div class="row" > <div class="col-md-12" style="text-align:center;" ><script async src="https://pagead2.googlesyndication.com/pagead/js/adsbygoogle.js"></script> <!-- 2) Display - Horizontal - Author - Responsive --> <ins class="adsbygoogle" style="display:block" data-ad-client="ca-pub-9333911690486629" data-ad-slot="2879047309" data-ad-format="auto" data-full-width-responsive="true"></ins> <script> (adsbygoogle = window.adsbygoogle || []).push({}); </script></div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/27940765/Management-of-Children-With-Hereditary-Angioedema-Due-to-C1-Inhibitor-Deficiency" >Management of Children With Hereditary Angioedema Due to C1 Inhibitor Deficiency.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Michael+M+Frank'>Michael M Frank</a> <a href='https://www.pubfacts.com/author/Bruce+Zuraw'>Bruce Zuraw</a> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/Timothy+Craig'>Timothy Craig</a> <a href='https://www.pubfacts.com/author/Paula+Busse'>Paula Busse</a> <a href='https://www.pubfacts.com/author/Sandra+Christiansen'>Sandra Christiansen</a> <a href='https://www.pubfacts.com/author/Marc+Davis-Lorton'>Marc Davis-Lorton</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Marc+Riedl'>Marc Riedl</a> <a href='https://www.pubfacts.com/author/+'> </a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Pediatrics 2016 11;138(5)</p><p>University of California Medical Center, San Diego, California.</p><div class="pubDescriptor" style="margin-top:20px;">Hereditary angioedema (HAE) is a potentially life-threatening inherited disease characterized by attacks of skin swelling, severe abdominal pain, and upper airway swelling. Attacks typically begin in childhood, but the appropriate diagnosis is often missed. Attacks do not respond to epinephrine, antihistamines, or glucocorticoids. Recently, many effective drugs have been approved for treatment of adults with HAE, and the Medical Advisory Board of the HAE Patient's Association has developed and reported treatment recommendations for adults. Only 1 medication is approved for treatment of children <12 years of age, and there are no reported consensus recommendations for treatment of young children in the United States. The 11-member Medical Advisory Board, with extensive experience in the treatment of children, in concert with the leaders of the HAE Patient's Association, has developed these consensus recommendations to help in recognition, diagnosis, treatment of attacks, and prophylaxis of children with HAE.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/27940765/Management-of-Children-With-Hereditary-Angioedema-Due-to-C1-Inhibitor-Deficiency" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1542/peds.2016-0575' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-27940765-n' >http://dx.doi.org/10.1542/peds.2016-0575</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> November 2016</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/27940765" target="_blank" title="" class="fb socialShare" id="fb-a-27940765"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/27940765" target="_blank" title="" class="tw socialShare" id="tw-a-27940765"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/27940765&title=Management+of+Children+With+Hereditary+Angioedema+Due+to+C1+Inhibitor+Deficiency.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-27940765"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/27931305/Subcutaneous-administration-of-human-C1-inhibitor-with-recombinant-human-hyaluronidase-in-patients-w" >Subcutaneous administration of human C1 inhibitor with recombinant human hyaluronidase in patients with hereditary angioedema.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/Murat+Ba'>Murat Ba</a> <a href='https://www.pubfacts.com/author/Janne+Bjrkander'>Janne Bjrkander</a> <a href='https://www.pubfacts.com/author/Markus+Magerl'>Markus Magerl</a> <a href='https://www.pubfacts.com/author/Marcus+Maurer'>Marcus Maurer</a> <a href='https://www.pubfacts.com/author/Kevin+Rockich'>Kevin Rockich</a> <a href='https://www.pubfacts.com/author/Hongzi+Chen'>Hongzi Chen</a> <a href='https://www.pubfacts.com/author/Jennifer+Schranz'>Jennifer Schranz</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Allergy Asthma Proc 2016 Nov;37(6):489-500</p><p>Division of Rheumatology, Allergy and Immunology, US HAEA Angioedema Center, University of CaliforniaSan Diego School of Medicine, La Jolla, California, USA.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: The currently approved method of C1 inhibitor (C1 INH) administration for patients with hereditary angioedema with C1 INH deficiency (HAE) is by intravenous injection. A C1 INH subcutaneous formulation may provide an attractive mode of administration for some patients.<BR><BR><em>Objective</em>: To evaluate efficacy and safety of two doses of subcutaneous, plasma-derived C1 INH with the dispersing agent, recombinant human hyaluronidase (rHuPH20) to prevent angioedema attacks in patients with HAE.<BR><BR><em>Methods</em>: A randomized, double-blind, dose-ranging, crossover study, patients 12 years of age (n = 47) with a confirmed diagnosis of HAE were randomly assigned to receive subcutaneous injections of 1000 U C1 INH with 24,000 U rHuPH20 or 2000 U C1 INH with 48,000 U rHuPH20 every 3 or 4 days for 8 weeks and then crossed-over for another 8-week period. The primary efficacy end point was the number of angioedema attacks during each treatment period.<BR><BR><em>Results</em>: The study was terminated early as a precaution related to non-neutralizing antibodies to rHuPH20 in 45% of patients. The mean standard deviation number of angioedema attacks during the 8-week treatment periods were 1.58 1.59 with 1000 U C1 INH and 0.97 1.26 with 2000 U. The mean (95% confidence interval [CI]) within-patient difference (2000 U-1000 U, respectively) was 0.61 (95% CI, 1.23 to 0.01) attacks per month (p = 0.0523), and 0.56 (95% CI, 1.06 to 0.05) attacks that required acute treatment, (p = 0.0315). No deaths or other serious adverse events were reported. Injection-site reaction was the most common adverse event.<BR><BR><em>Conclusion</em>: Despite early termination, this study demonstrated a clinically and statistically significant difference in burden of disease, which favored 2000 U C1 INH, without associated serious adverse events.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/27931305/Subcutaneous-administration-of-human-C1-inhibitor-with-recombinant-human-hyaluronidase-in-patients-w" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.2500/aap.2016.37.4006' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-27931305-n' >http://dx.doi.org/10.2500/aap.2016.37.4006</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> November 2016</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/27931305" target="_blank" title="" class="fb socialShare" id="fb-a-27931305"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/27931305" target="_blank" title="" class="tw socialShare" id="tw-a-27931305"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/27931305&title=Subcutaneous+administration+of+human+C1+inhibitor+with+recombinant+human+hyaluronidase+in+patients+with+hereditary+angioedema.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-27931305"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/27818136/Subcutaneous-Icatibant-for-the-Treatment-of-Hereditary-Angioedema-Attacks-Comparison-of-Home-Self-Ad" >Subcutaneous Icatibant for the Treatment of Hereditary Angioedema Attacks: Comparison of Home Self-Administration with Administration at a Medical Facility.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Iris+M+Otani'>Iris M Otani</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Shelley+Hurwitz'>Shelley Hurwitz</a> <a href='https://www.pubfacts.com/author/Huamin+Henry+Li'>Huamin Henry Li</a> <a href='https://www.pubfacts.com/author/Timothy+J+Craig'>Timothy J Craig</a> <a href='https://www.pubfacts.com/author/Niki+S+Holtzman'>Niki S Holtzman</a> <a href='https://www.pubfacts.com/author/Matthew+I+Iandoli'>Matthew I Iandoli</a> <a href='https://www.pubfacts.com/author/Julie+Tucker'>Julie Tucker</a> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> <a href='https://www.pubfacts.com/author/Bruce+L+Zuraw'>Bruce L Zuraw</a> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>J Allergy Clin Immunol Pract 2017 Mar - Apr;5(2):442-447.e1. Epub 2016 Nov 3.</p><p>Division of Rheumatology, Allergy, and Immunology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Mass.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: Hereditary angioedema (HAE) is a life-threatening disorder characterized by recurrent angioedema. Icatibant, a subcutaneous bradykinin-B2-receptor antagonist, is an effective on-demand therapy. Data outside the United States suggest that self-administration is tolerated and patient-preferred compared with administration by health care professionals at medical facilities (HCP-administration).<BR><BR><em>Objective</em>: A prospective, multicenter study was conducted in the United States to compare icatibant self-administration and HCP-administration.<BR><BR><em>Methods</em>: Subjects 18 years or older with type I or II HAE were recruited. The first 2 HAE attacks after enrollment were treated at medical facilities. Subjects were instructed by a health care professional on self-administration during icatibant treatment for the second HAE attack. Icatibant was self-administered for all subsequent attacks. For each treated HAE attack, efficacy, safety, and tolerability data were recorded.<BR><BR><em>Results</em>: Nineteen patients with HAE received icatibant for 79 distinct HAE attacks. Mean attack duration was significantly shorter with self-administration (n = 50; 547 ± 510 minutes) than with HCP-administration (n = 29; 968 ± 717 minutes; P = .006). Mean time to treatment was significantly shorter with self-administration (143 ± 226 minutes) than with HCP-administration (361 ± 503 minutes; P < .0001). Shorter times to treatment were associated with shorter time from treatment to symptom resolution (r = 0.35; P = .02). Improvements in visual analog scale score and patient symptom score from pretreatment to 4 hours postinjection were comparable between self-administration and HCP-administration. There were no serious adverse events or discontinuations due to adverse events with self-administration or HCP-administration.<BR><BR><em>Conclusions</em>: Icatibant self-administration shortened attack duration and time to treatment, with no difference in safety or local tolerability compared with HCP-administration. These findings support icatibant as an effective on-demand option for home-based treatment.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/27818136/Subcutaneous-Icatibant-for-the-Treatment-of-Hereditary-Angioedema-Attacks-Comparison-of-Home-Self-Ad" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.jaip.2016.09.023' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-27818136-n' >http://dx.doi.org/10.1016/j.jaip.2016.09.023</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> November 2017</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/27818136" target="_blank" title="" class="fb socialShare" id="fb-a-27818136"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/27818136" target="_blank" title="" class="tw socialShare" id="tw-a-27818136"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/27818136&title=Subcutaneous+Icatibant+for+the+Treatment+of+Hereditary+Angioedema+Attacks%3A+Comparison+of+Home+Self-Administration+with+Administration+at+a+Medical+Facility.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-27818136"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/26556097/Icatibant-for-Multiple-Hereditary-Angioedema-Attacks-across-the-Controlled-and-Open-Label-Extension-" >Icatibant for Multiple Hereditary Angioedema Attacks across the Controlled and Open-Label Extension Phases of FAST-3.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Henriette+Farkas'>Henriette Farkas</a> <a href='https://www.pubfacts.com/author/Dumitru+Moldovan'>Dumitru Moldovan</a> <a href='https://www.pubfacts.com/author/Elias+Toubi'>Elias Toubi</a> <a href='https://www.pubfacts.com/author/Jovanna+Baptista'>Jovanna Baptista</a> <a href='https://www.pubfacts.com/author/Timothy+Craig'>Timothy Craig</a> <a href='https://www.pubfacts.com/author/Marc+Riedl'>Marc Riedl</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Int Arch Allergy Immunol 2015 11;168(1):44-55. Epub 2015 Nov 11.</p><p>AARA Research Center, Dallas, Tex., USA.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: In randomized, controlled, double-blind, multicenter phase 3 studies, one icatibant injection was efficacious and generally well tolerated in patients with a single hereditary angioedema (HAE) attack. Here, the efficacy and safety of icatibant for multiple HAE attacks was evaluated across the controlled and open-label extension phases of the For Angioedema Subcutaneous Treatment (FAST)-3 study (NCT00912093).<BR><BR><em>Methods</em>: In the controlled phase, adults with HAE type I or II were randomized (1:1) to receive a single subcutaneous injection of icatibant 30 mg or placebo within 6 h of an attack becoming mild (laryngeal) or moderate (cutaneous/abdominal). Open-label icatibant was administered for severe laryngeal symptoms. In the open-label extension phase, patients could receive up to three icatibant injections per attack. Efficacy and safety were analyzed for the first five icatibant-treated attacks at any location (prospective analysis) and laryngeal attacks (post hoc analysis) across both phases. Efficacy outcomes were based on patient-reported symptom severity (visual analog scale).<BR><BR><em>Results</em>: In groups of patients with one to five icatibant-treated attacks at any location (n = 88), the median times to onset of symptom relief, onset of primary symptom relief and almost complete symptom relief were 1.9-2.1, 1.5-2.0 and 3.5-19.7 h, respectively. The same outcomes for laryngeal attacks (n = 25) were 1.0-2.0, 1.0-2.0 and 1.5-8.1 h, respectively. The most frequently reported adverse events were a worsening or recurrence of HAE attack, headache and nasopharyngitis. Two serious adverse events (arrhythmia and noncardiac chest pain) were considered to be related to icatibant.<BR><BR><em>Conclusions</em>: Icatibant was efficacious and generally well tolerated across multiple HAE attacks, including laryngeal attacks.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/26556097/Icatibant-for-Multiple-Hereditary-Angioedema-Attacks-across-the-Controlled-and-Open-Label-Extension-" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1159/000441060' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-26556097-n' >http://dx.doi.org/10.1159/000441060</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> February 2016</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/26556097" target="_blank" title="" class="fb socialShare" id="fb-a-26556097"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/26556097" target="_blank" title="" class="tw socialShare" id="tw-a-26556097"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/26556097&title=Icatibant+for+Multiple+Hereditary+Angioedema+Attacks+across+the+Controlled+and+Open-Label+Extension+Phases+of+FAST-3.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-26556097"><i class="icon-linkedin"></i></a> </div></div> </div> </div> <div class="row" > <div class="col-md-12" style="text-align:center;" ><script async src="https://pagead2.googlesyndication.com/pagead/js/adsbygoogle.js"></script> <!-- 2) Display - Horizontal - Author - Responsive --> <ins class="adsbygoogle" style="display:block" data-ad-client="ca-pub-9333911690486629" data-ad-slot="2879047309" data-ad-format="auto" data-full-width-responsive="true"></ins> <script> (adsbygoogle = window.adsbygoogle || []).push({}); </script></div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/25803135/Prevalence-of-hereditary-angioedema-in-untested-first-degree-blood-relatives-of-known-subjects-with-" >Prevalence of hereditary angioedema in untested first-degree blood relatives of known subjects with hereditary angioedema.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Marc+A+Riedl'>Marc A Riedl</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Paula+Busse'>Paula Busse</a> <a href='https://www.pubfacts.com/author/Howard+Levy'>Howard Levy</a> <a href='https://www.pubfacts.com/author/Tamara+Steele'>Tamara Steele</a> <a href='https://www.pubfacts.com/author/Jeffrey+Dayno'>Jeffrey Dayno</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Allergy Asthma Proc 2015 May-Jun;36(3):206-12. Epub 2015 Mar 23.</p><p>University of California-San Diego School of Medicine, La Jolla, CA, USA.</p><div class="pubDescriptor" style="margin-top:20px;">Hereditary angioedema (HAE) is a rare autosomal-dominant disease characterized by recurring attacks of nonpruritic, nonpitting edema caused by an inherited deficiency or dysfunction of C1 esterase inhibitor (C1 INH). Symptoms can present years before an accurate diagnosis is made. The objective of this study, the Angioedema Clinical Epidemiology Testing Initiative for the Study of Hereditary Angioedema, was to determine the prevalence and clinical manifestations of HAE in untested first-degree blood relatives of known patients with HAE. Patients with a confirmed diagnosis of HAE recruited first-degree relatives who had not been evaluated for HAE. Enrolled subjects underwent complement testing (C4, C1 INH antigen, and functional C1 INH). If the lab tests were abnormal, the enrolled subjects returned to the site for a follow-up visit and questionnaire. Overall, 31 patients with HAE and 46 first-degree relatives enrolled in the study. Of 46 enrolled relatives, 30 (65%) had lab test results that ruled out a diagnosis of HAE, two (4%) were categorized as "HAE not ruled out," and 14 (30%) were newly diagnosed with HAE. Of 14 newly diagnosed subjects, nine (64%) reported having experienced symptoms that may have been related to HAE, such as swelling in the throat, face, or extremities or abdominal pain. When reported, median age of symptom onset in these 14 subjects was nine years whereas newly diagnosed asymptomatic subjects had a median chronological age of six years. These 14 subjects reported a historic mean standard deviation rate of 2.51 (5.59) swelling episodes per month with a mean standard deviation duration of 1.6 (0.74) days. This study's findings reinforce the importance of testing family members of patients with HAE to detect this hereditary condition. </div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/25803135/Prevalence-of-hereditary-angioedema-in-untested-first-degree-blood-relatives-of-known-subjects-with-" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.2500/aap.2015.36.3833' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-25803135-n' >http://dx.doi.org/10.2500/aap.2015.36.3833</a></td><td>DOI Listing</td></tr><tr><td><i class='icon-doc'></i><a href='http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4405600' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_PMC", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-25803135-n'>http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4405600</a></td><td>PMC</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> February 2016</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/25803135" target="_blank" title="" class="fb socialShare" id="fb-a-25803135"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/25803135" target="_blank" title="" class="tw socialShare" id="tw-a-25803135"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/25803135&title=Prevalence+of+hereditary+angioedema+in+untested+first-degree+blood+relatives+of+known+subjects+with+hereditary+angioedema.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-25803135"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/25609336/Comparison-of-chromogenic-and-ELISA-functional-C1-inhibitor-tests-in-diagnosing-hereditary-angioedem" >Comparison of chromogenic and ELISA functional C1 inhibitor tests in diagnosing hereditary angioedema.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/Paula+Busse'>Paula Busse</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Ashley+Frazer-Abel'>Ashley Frazer-Abel</a> <a href='https://www.pubfacts.com/author/Howard+Levy'>Howard Levy</a> <a href='https://www.pubfacts.com/author/Tamara+Steele'>Tamara Steele</a> <a href='https://www.pubfacts.com/author/Jeffrey+Dayno'>Jeffrey Dayno</a> <a href='https://www.pubfacts.com/author/Marc+Riedl'>Marc Riedl</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>J Allergy Clin Immunol Pract 2015 Mar-Apr;3(2):200-5. Epub 2014 Oct 11.</p><p>Department of Medicine, Division of Rheumatology, Allergy and Immunology, University of California-San Diego, La Jolla, Calif.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Introduction</em>: Measuring functional C1 inhibitor (C1 INH) with chromogenic or ELISA methods can confirm a diagnosis of hereditary angioedema (HAE) due to C1 INH deficiency. Previous studies found differences in the agreement of these assays.<BR><BR><em>Objective</em>: To evaluate the agreement between chromogenic or ELISA methods in the context of an observational study.<BR><BR><em>Methods</em>: Patients with previously confirmed HAE underwent functional C1 INH testing. These patients contacted first-degree relatives (parents, siblings, or offspring) not previously evaluated for HAE, who underwent a panel of complement testing, including for functional C1 INH.<BR><BR><em>Results</em>: Overall, 31 patients with HAE and 46 untested relatives enrolled. Of 46 relatives, 14 (30.4%) were newly diagnosed with HAE based on their laboratory results. Among the 31 patients previously confirmed with HAE, all had low functional C1 INH according to the chromogenic method, whereas 22 (71.0%) had low, 7 (22.6%) had equivocal, and 2 (6.5%) had normal functional C1 INH according to the ELISA method. In the 14 newly diagnosed relatives, all had low functional C1 INH according to the chromogenic method, whereas 11 (78.5%) had low and 3 (21.4%) had equivocal results according to the ELISA method.<BR><BR><em>Conclusion</em>: Despite the apparent discordance in the ELISA and chromogenic assays in a small number of patients, both were useful in measuring functional C1 INH. To establish the diagnosis of HAE due to C1 INH deficiency, functional C1 INH results should be interpreted in combination with family and clinical history, and with other complement tests.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/25609336/Comparison-of-chromogenic-and-ELISA-functional-C1-inhibitor-tests-in-diagnosing-hereditary-angioedem" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.jaip.2014.08.002' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-25609336-n' >http://dx.doi.org/10.1016/j.jaip.2014.08.002</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> December 2015</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/25609336" target="_blank" title="" class="fb socialShare" id="fb-a-25609336"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/25609336" target="_blank" title="" class="tw socialShare" id="tw-a-25609336"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/25609336&title=Comparison+of+chromogenic+and+ELISA+functional+C1+inhibitor+tests+in+diagnosing+hereditary+angioedema.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-25609336"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/25609335/Characterization-of-anaphylaxis-after-ecallantide-treatment-of-hereditary-angioedema-attacks" >Characterization of anaphylaxis after ecallantide treatment of hereditary angioedema attacks.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Timothy+J+Craig'>Timothy J Craig</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/Marc+Riedl'>Marc Riedl</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Andrew+J+MacGinnitie'>Andrew J MacGinnitie</a> <a href='https://www.pubfacts.com/author/Leslie+E+Stolz'>Leslie E Stolz</a> <a href='https://www.pubfacts.com/author/Joseph+Biedenkapp'>Joseph Biedenkapp</a> <a href='https://www.pubfacts.com/author/Yung+Chyung'>Yung Chyung</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>J Allergy Clin Immunol Pract 2015 Mar-Apr;3(2):206-212.e4. Epub 2014 Oct 29.</p><p>Dyax Corp., Burlington, Mass.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: Ecallantide is a human plasma kallikrein inhibitor indicated for treatment of acute attacks of hereditary angioedema for patients 12 years of age and older. Ecallantide is produced in Pichia pastoris yeast cells by recombinant DNA technology. Use of ecallantide has been associated with a risk of hypersensitivity reactions, including anaphylaxis.<BR><BR><em>Objective</em>: The objective of this detailed retrospective data review was to characterize anaphylaxis cases within the ecallantide clinical trials database.<BR><BR><em>Methods</em>: Potential cases of hypersensitivity reactions in the ecallantide clinical development program were identified by examining reported adverse events. The National Institute of Allergy and Infectious Disease criteria were used to identify those events that were consistent with anaphylaxis; these cases were then reviewed in detail. Results from investigational antibody testing also were examined.<BR><BR><em>Results</em>: Among patients who received subcutaneous ecallantide (n = 230 patients; 1045 doses of 30 mg ecallantide), 8 patients (3.5%) had reactions that met the National Institute of Allergy and Infectious Disease criteria for anaphylaxis; none occurred on first exposure to the drug. All 8 reactions had symptom onset within 1 hour of exposure and cutaneous manifestations commonly observed in type I hypersensitivity reactions. All the reactions responded to standard management of type I hypersensitivity reactions and resolved without fatal outcomes. IgE antibody testing to ecallantide or P pastoris was not consistently positive in patients who experienced apparent type I hypersensitivity reactions.<BR><BR><em>Conclusion</em>: Anaphylaxis episodes after subcutaneous ecallantide exposure have clinical features suggestive of type I hypersensitivity reactions. However, anti-ecallantide or anti-P pastoris IgE antibody status was not found to be reliably associated with anaphylaxis.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/25609335/Characterization-of-anaphylaxis-after-ecallantide-treatment-of-hereditary-angioedema-attacks" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.jaip.2014.09.001' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-25609335-n' >http://dx.doi.org/10.1016/j.jaip.2014.09.001</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> December 2015</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/25609335" target="_blank" title="" class="fb socialShare" id="fb-a-25609335"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/25609335" target="_blank" title="" class="tw socialShare" id="tw-a-25609335"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/25609335&title=Characterization+of+anaphylaxis+after+ecallantide+treatment+of+hereditary+angioedema+attacks.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-25609335"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/25295804/Quality-of-life-in-patients-with-hereditary-angioedema-receiving-therapy-for-routine-prevention-of-a" >Quality of life in patients with hereditary angioedema receiving therapy for routine prevention of attacks.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Dave+P+Miller'>Dave P Miller</a> <a href='https://www.pubfacts.com/author/Scott+Newcomer'>Scott Newcomer</a> <a href='https://www.pubfacts.com/author/David+Fitts'>David Fitts</a> <a href='https://www.pubfacts.com/author/Jeffrey+Dayno'>Jeffrey Dayno</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Allergy Asthma Proc 2014 Sep-Oct;35(5):371-6</p><p>Allergy and Asthma Research Associates, Dallas, Texas, USA.</p><div class="pubDescriptor" style="margin-top:20px;">Patients with hereditary angioedema (HAE) have impaired health-related quality of life (HRQoL), but the effect of preventative treatment strategies on HRQoL has not been evaluated. This study was designed to evaluate the effect of routine prevention therapy with nanofiltered C1 inhibitor (C1 INH-nf; human) on the HRQoL of patients with HAE. Thiry-six-item Short Form (SF-36) Version 1.0 questionnaires were administered at the beginning and end of two 12-week treatment periods in this multicenter, randomized, placebo-controlled, crossover study. Patients (n = 22) received intravenous injections of 1000 U of C1 INH-nf or placebo every 3-4 days for 12 weeks and then crossed over to the other treatment arm for a second 12-week period. Patients could receive open-label C1 INH-nf (1000 U) for the acute treatment of angioedema attacks in either arm of the study. Sixteen patients had evaluable SF-36 data. Mean physical component summary scores (PCSs) were 36.41 at baseline, 37.06 at the end of the placebo period, and 43.92 at the end of the C1 INH-nf period. Mean mental component summary scores (MCSs) were 49.90, 44.98, and 54.00, respectively. Least square mean differences (95% confidence intervals) between C1 INH-nf and placebo in norm-based SF-36 scores at the end of each treatment period were 6.55 (1.48, 11.62; p = 0.015) for PCS and 8.70 (1.67, 15.72; p = 0.019) for MCS. In a clinical trial setting, patients with HAE had significantly better HRQoL after 12 weeks of C1 INH-nf for routine prevention compared with acute treatment of individual angioedema attacks in the absence of routine prevention while on placebo. This study was a part of the clinical trial NCT01005888 registered in www.clinicaltrials.gov. </div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/25295804/Quality-of-life-in-patients-with-hereditary-angioedema-receiving-therapy-for-routine-prevention-of-a" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.2500/aap.2014.35.3783' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-25295804-n' >http://dx.doi.org/10.2500/aap.2014.35.3783</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> June 2015</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/25295804" target="_blank" title="" class="fb socialShare" id="fb-a-25295804"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/25295804" target="_blank" title="" class="tw socialShare" id="tw-a-25295804"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/25295804&title=Quality+of+life+in+patients+with+hereditary+angioedema+receiving+therapy+for+routine+prevention+of+attacks.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-25295804"><i class="icon-linkedin"></i></a> </div></div> </div> </div> <div class="row" > <div class="col-md-12" style="text-align:center;" ><script async src="https://pagead2.googlesyndication.com/pagead/js/adsbygoogle.js"></script> <!-- 2) Display - Horizontal - Author - Responsive --> <ins class="adsbygoogle" style="display:block" data-ad-client="ca-pub-9333911690486629" data-ad-slot="2879047309" data-ad-format="auto" data-full-width-responsive="true"></ins> <script> (adsbygoogle = window.adsbygoogle || []).push({}); </script></div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/25198193/Treatment-of-hereditary-angioedema-with-icatibant-efficacy-in-clinical-trials-versus-effectiveness-i" >Treatment of hereditary angioedema with icatibant: efficacy in clinical trials versus effectiveness in the real-world setting.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Marcus+Maurer'>Marcus Maurer</a> <a href='https://www.pubfacts.com/author/Hilary+J+Longhurst'>Hilary J Longhurst</a> <a href='https://www.pubfacts.com/author/Vincent+Fabien'>Vincent Fabien</a> <a href='https://www.pubfacts.com/author/H+Henry+Li'>H Henry Li</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Allergy Asthma Proc 2014 Sep-Oct;35(5):377-81. Epub 2014 Aug 6.</p><p>Department of Dermatology and Allergy, Allergie-Centrum-Charité, Charité-Universitätsmedizin Berlin, Berlin, Germany.</p><div class="pubDescriptor" style="margin-top:20px;">Icatibant was efficacious and generally well tolerated for type I or II hereditary angioedema (HAE) attacks in adults in the phase III, randomized, placebo-controlled For Angioedema Subcutaneous Treatment (FAST)-3 trial. The Icatibant Outcome Survey (IOS) is an international, observational study assessing icatibant treatment of HAE attacks. We conducted a posthoc analysis to compare for the first time the treatment of HAE type I or II attacks in patients prescribed icatibant in real-world (IOS) versus controlled trial settings (FAST-3). In FAST-3, patients received icatibant administered by health care professionals (HCPs). In IOS, patients self-administered icatibant or were treated by HCPs. Median time to treatment, time to resolution (almost complete resolution [FAST-3] or complete resolution [IOS]), and attack duration in patients who were treated by an HCP were compared between IOS and FAST-3. Descriptive statistical methods compared nonlaryngeal attacks treated less than 12 hours from attack onset. Analysis included 102 patients (376 attacks) from IOS and 43 patients (43 attacks) from FAST-3 (controlled phase). All endpoints were significantly longer for patients in FAST-3 (HCP administration) versus IOS (HCP administration) (p < .001; all comparisons). For FAST-3 (HCP administered) versus IOS (HCP administered), median time from attack onset to treatment was 6.5 versus 2.0 hours, median time to symptom resolution was 8.0 versus 3.5 hours, and median attack duration was 16.9 versus 7.3 hours, respectively. For combined HCP and self-administration in IOS, these endpoints were 1.6, 4.4, and 7.8 hours, respectively. This posthoc analysis showed for the first time that type I and II HAE attacks were treated earlier with icatibant in a real-world versus a phase III setting, with a shortened time to symptom resolution and attack duration. ClinicalTrials.gov NCT00912093 (FAST-3); NCT01034969 (IOS). </div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/25198193/Treatment-of-hereditary-angioedema-with-icatibant-efficacy-in-clinical-trials-versus-effectiveness-i" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.2500/aap.2014.35.3780' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-25198193-n' >http://dx.doi.org/10.2500/aap.2014.35.3780</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> June 2015</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/25198193" target="_blank" title="" class="fb socialShare" id="fb-a-25198193"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/25198193" target="_blank" title="" class="tw socialShare" id="tw-a-25198193"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/25198193&title=Treatment+of+hereditary+angioedema+with+icatibant%3A+efficacy+in+clinical+trials+versus+effectiveness+in+the+real-world+setting.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-25198193"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/24730413/A-consensus-parameter-for-the-evaluation-and-management-of-angioedema-in-the-emergency-department" >A consensus parameter for the evaluation and management of angioedema in the emergency department.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Joseph+J+Moellman'>Joseph J Moellman</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/Christopher+Lindsell'>Christopher Lindsell</a> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> <a href='https://www.pubfacts.com/author/Paula+J+Busse'>Paula J Busse</a> <a href='https://www.pubfacts.com/author/Carlos+A+Camargo'>Carlos A Camargo</a> <a href='https://www.pubfacts.com/author/Sean+P+Collins'>Sean P Collins</a> <a href='https://www.pubfacts.com/author/Timothy+J+Craig'>Timothy J Craig</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Richard+Nowak'>Richard Nowak</a> <a href='https://www.pubfacts.com/author/Jesse+M+Pines'>Jesse M Pines</a> <a href='https://www.pubfacts.com/author/Ali+S+Raja'>Ali S Raja</a> <a href='https://www.pubfacts.com/author/Marc+Riedl'>Marc Riedl</a> <a href='https://www.pubfacts.com/author/Michael+J+Ward'>Michael J Ward</a> <a href='https://www.pubfacts.com/author/Bruce+L+Zuraw'>Bruce L Zuraw</a> <a href='https://www.pubfacts.com/author/Deborah+Diercks'>Deborah Diercks</a> <a href='https://www.pubfacts.com/author/Brian+Hiestand'>Brian Hiestand</a> <a href='https://www.pubfacts.com/author/Ronna+L+Campbell'>Ronna L Campbell</a> <a href='https://www.pubfacts.com/author/Sandra+Schneider'>Sandra Schneider</a> <a href='https://www.pubfacts.com/author/Richard+Sinert'>Richard Sinert</a> <a href='https://www.pubfacts.com/author/+'> </a> <a href='https://www.pubfacts.com/author/+'> </a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>Acad Emerg Med 2014 Apr;21(4):469-84</p><p>The Department of Emergency Medicine, University of Cincinnati College of Medicine, Cincinnati, OH.</p><div class="pubDescriptor" style="margin-top:20px;">Despite its relatively common occurrence and life-threatening potential, the management of angioedema in the emergency department (ED) is lacking in terms of a structured approach. It is paramount to distinguish the different etiologies of angioedema from one another and more specifically differentiate histaminergic-mediated angioedema from bradykinin-mediated angioedema, especially in lieu of the more novel treatments that have recently become available for bradykinin-mediated angioedema. With this background in mind, this consensus parameter for the evaluation and management of angioedema attempts to provide a working framework for emergency physicians (EPs) in approaching the patient with angioedema in terms of diagnosis and management in the ED. This consensus parameter was developed from a collaborative effort among a group of EPs and leading allergists with expertise in angioedema. After rigorous debate, review of the literature, and expert opinion, the following consensus guideline document was created. The document has been endorsed by the American College of Allergy, Asthma & Immunology (ACAAI) and the Society for Academic Emergency Medicine (SAEM). </div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/24730413/A-consensus-parameter-for-the-evaluation-and-management-of-angioedema-in-the-emergency-department" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1111/acem.12341' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-24730413-n' >http://dx.doi.org/10.1111/acem.12341</a></td><td>DOI Listing</td></tr><tr><td><i class='icon-doc'></i><a href='http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4100605' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_PMC", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-24730413-n'>http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4100605</a></td><td>PMC</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> April 2014</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/24730413" target="_blank" title="" class="fb socialShare" id="fb-a-24730413"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/24730413" target="_blank" title="" class="tw socialShare" id="tw-a-24730413"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/24730413&title=A+consensus+parameter+for+the+evaluation+and+management+of+angioedema+in+the+emergency+department.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-24730413"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/24565773/Escalating-doses-of-C1-esterase-inhibitor-CINRYZE-for-prophylaxis-in-patients-with-hereditary-angioe" >Escalating doses of C1 esterase inhibitor (CINRYZE) for prophylaxis in patients with hereditary angioedema.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/Michael+E+Manning'>Michael E Manning</a> <a href='https://www.pubfacts.com/author/Henry+Li'>Henry Li</a> <a href='https://www.pubfacts.com/author/Martha+V+White'>Martha V White</a> <a href='https://www.pubfacts.com/author/James+Baker'>James Baker</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Mark+A+Davis-Lorton'>Mark A Davis-Lorton</a> <a href='https://www.pubfacts.com/author/Kraig+W+Jacobson'>Kraig W Jacobson</a> <a href='https://www.pubfacts.com/author/Richard+G+Gower'>Richard G Gower</a> <a href='https://www.pubfacts.com/author/Colin+Broom'>Colin Broom</a> <a href='https://www.pubfacts.com/author/David+Fitts'>David Fitts</a> <a href='https://www.pubfacts.com/author/Jennifer+Schranz'>Jennifer Schranz</a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>J Allergy Clin Immunol Pract 2014 Jan-Feb;2(1):77-84. Epub 2013 Nov 11.</p><p>Clinical Research for Broom and Schranz and Biostatistics for Fitts, ViroPharma Incorporated, Exton, Pa.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: Nanofiltered C1 inhibitor (human) is approved in the United States for routine prophylaxis of angioedema attacks in patients with hereditary angioedema, a rare disease caused by a deficiency of functional C1 inhibitor.<BR><BR><em>Objective</em>: To assess the safety of escalating doses of nanofiltered C1 inhibitor (human) in patients who were not adequately controlled on the indicated dose (1000 U every 3 or 4 days).<BR><BR><em>Methods</em>: Eligible patients had >1 attack/month over the 3 months before the trial. Doses were escalated to 1500 U every 3 or 4 days for 12 weeks, at which point, the patients were evaluated. If treatment was successful (≤1 attack/mo) or at the investigator's discretion, the patients entered a 3-month follow-up period. The patients with an average of >1 attack/month were eligible for further escalation to 2000 U and then 2500 U.<BR><BR><em>Results</em>: Twenty patients started at 1500 U; 13 were escalated to 2000 U, and 12 were escalated to 2500 U. Eighteen patients reported adverse events. Two patients reported 4 serious adverse events (cerebral cystic hygroma, laryngeal angioedema attack, anemia, and bile duct stone) that were considered by investigators to be unrelated to treatment. Notably, there were no systemic thrombotic events or discontinuations due to adverse events. Fourteen patients were treated successfully (70%), continued to the follow-up period at the investigator's discretion, or experienced a reduction in attacks of >1.0/month.<BR><BR><em>Conclusions</em>: Dose escalation of nanofiltered C1 inhibitor (human) up to 2500 U was well tolerated and reduced attack frequency in the majority of patients.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/24565773/Escalating-doses-of-C1-esterase-inhibitor-CINRYZE-for-prophylaxis-in-patients-with-hereditary-angioe" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.jaip.2013.09.008' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-24565773-n' >http://dx.doi.org/10.1016/j.jaip.2013.09.008</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> April 2014</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/24565773" target="_blank" title="" class="fb socialShare" id="fb-a-24565773"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/24565773" target="_blank" title="" class="tw socialShare" id="tw-a-24565773"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/24565773&title=Escalating+doses+of+C1+esterase+inhibitor+%28CINRYZE%29+for+prophylaxis+in+patients+with+hereditary+angioedema.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-24565773"><i class="icon-linkedin"></i></a> </div></div> </div> </div><div class="publication-block"><h3><a href="https://www.pubfacts.com/detail/24565617/US-Hereditary-Angioedema-Association-Medical-Advisory-Board-2013-recommendations-for-the-management-" >US Hereditary Angioedema Association Medical Advisory Board 2013 recommendations for the management of hereditary angioedema due to C1 inhibitor deficiency.</a></h3> <div class="publication-auther"> <h5>Authors:</h5> <a href='https://www.pubfacts.com/author/Bruce+L+Zuraw'>Bruce L Zuraw</a> <a href='https://www.pubfacts.com/author/Aleena+Banerji'>Aleena Banerji</a> <a href='https://www.pubfacts.com/author/Jonathan+A+Bernstein'>Jonathan A Bernstein</a> <a href='https://www.pubfacts.com/author/Paula+J+Busse'>Paula J Busse</a> <a href='https://www.pubfacts.com/author/Sandra+C+Christiansen'>Sandra C Christiansen</a> <a href='https://www.pubfacts.com/author/Mark+Davis-Lorton'>Mark Davis-Lorton</a> <a href='https://www.pubfacts.com/author/Michael+M+Frank'>Michael M Frank</a> <a href='https://www.pubfacts.com/author/Henry+H+Li'>Henry H Li</a> <a href='https://www.pubfacts.com/author/William+R+Lumry'>William R Lumry</a> <a href='https://www.pubfacts.com/author/Marc+Riedl'>Marc Riedl</a> <a href='https://www.pubfacts.com/author/+'> </a> </div> <div class="publication-dtl clearfix"> <div class="publication-dtl-lft fl"><p>J Allergy Clin Immunol Pract 2013 Sep-Oct;1(5):458-67. Epub 2013 Aug 30.</p><p>Clinical Immunology and Allergy, Department of Medicine, University of California Los Angeles, Los Angeles, Calif.</p><div class="pubDescriptor" style="margin-top:20px;"><em>Background</em>: The treatment of hereditary angioedema (HAE) has undergone dramatic changes as newer medicines have become available in recent years. Optimal care of these patients requires a comprehensive management plan. Although several consensus papers have been published concerning the diagnosis and treatment of HAE, guidelines for a comprehensive management plan have not been developed.<BR><BR><em>Objective</em>: To develop state-of-the-art recommendations for the treatment and management of HAE due to C1 inhibitor (C1INH) deficiency in the United States.<BR><BR><em>Methods</em>: Members of the US Hereditary Angioedema Association Medical Advisory Board began by reviewing the literature concerning treatment of HAE. Preliminary recommendations were developed based on the literature review, discussions in a face-to-face meeting, and refinements in a series of drafts. Final recommendations reflect the unanimous consensus of the medical advisory board and the US Hereditary Angioedema Association leadership.<BR><BR><em>Results</em>: Recommendations are provided regarding a comprehensive care plan for HAE, including the following: development of an overall management plan, treatment of angioedema attacks, prophylactic treatment, and patient monitoring.<BR><BR><em>Conclusion</em>: A comprehensive individualized management plan developed between an expert HAE physician and the patient, in collaboration with local medical providers and emergency departments, can provide patients with the best opportunity to lead a normal life.</div><div class="recent-author-more view-article"> <a href="https://www.pubfacts.com/detail/24565617/US-Hereditary-Angioedema-Association-Medical-Advisory-Board-2013-recommendations-for-the-management-" class="btn btn-default btn-labeled"><span class="btn-label"><i class="icon-book-open"></i></span>View Article and Find Full Text PDF</a> </div><table class="table table-bordered table-striped downloadTable" > <thead > <tr> <th ><h3>Download full-text PDF</h3></th> <th >Source</th> </tr></thead> <tbody><tr><td><i class='icon-doc'></i><a href='http://dx.doi.org/10.1016/j.jaip.2013.07.002' rel='nofollow' target='_blank' onClick='ga("send", "event", "fulltextclick_DOI Listing", "click", "article");' style='text-decoration:underline;' class='clickTrack' data-ref='fulltext-24565617-n' >http://dx.doi.org/10.1016/j.jaip.2013.07.002</a></td><td>DOI Listing</td></tr></tbody></table></div><div class="publication-dtl-rgt fr"><div class="publish-date"> January 2015</div><div class="publication-social"><a href="https://www.facebook.com/sharer/sharer.php?u=https://www.pubfacts.com/detail/24565617" target="_blank" title="" class="fb socialShare" id="fb-a-24565617"><i class="icon-facebook"></i></a> <a href="https://twitter.com/intent/tweet?text=Full+Article+on+PubFacts%3A&url=https://www.pubfacts.com/detail/24565617" target="_blank" title="" class="tw socialShare" id="tw-a-24565617"><i class="icon-twitter"></i></a> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https://www.pubfacts.com/detail/24565617&title=US+Hereditary+Angioedema+Association+Medical+Advisory+Board+2013+recommendations+for+the+management+of+hereditary+angioedema+due+to+C1+inhibitor+deficiency.&summary=Article+Summary+on+PubFacts.com" target="_blank" title="" class="in socialShare" id="in-a-24565617"><i class="icon-linkedin"></i></a> </div></div> </div> </div> <div class="row" > <div class="col-md-12" style="text-align:center;" ><script async src="https://pagead2.googlesyndication.com/pagead/js/adsbygoogle.js"></script> <!-- 2) Display - Horizontal - Author - Responsive --> <ins class="adsbygoogle" style="display:block" data-ad-client="ca-pub-9333911690486629" data-ad-slot="2879047309" data-ad-format="auto" data-full-width-responsive="true"></ins> <script> (adsbygoogle = window.adsbygoogle || []).push({}); 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