Publications by authors named "Sherif M Badawy"

93 Publications

Identifying barriers to evidence-based care for sickle cell disease: results from the Sickle Cell Disease Implementation Consortium cross-sectional survey of healthcare providers in the USA.

BMJ Open 2021 Nov 17;11(11):e050880. Epub 2021 Nov 17.

Department of Hematology, St Jude Children's Research Hospital, Memphis, Tennessee, USA.

Objectives: Sickle cell disease (SCD) leads to chronic and acute complications that require specialised care to manage symptoms and optimise clinical results. The National Heart Lung and Blood Institute (NHLBI) evidence-based guidelines assist providers in caring for individuals with SCD, but adoption of these guidelines by providers has not been optimal. The objective of this study was to identify barriers to treating individuals with SCD.

Methods: The SCD Implementation Consortium aimed to investigate the perception and level of comfort of providers regarding evidence-based care by surveying providers in the regions of six clinical centres across the USA, focusing on non-emergency care from the providers' perspective.

Results: Respondents included 105 providers delivering clinical care for individuals with SCD. Areas of practice were most frequently paediatrics (24%) or haematology/SCD specialist (24%). The majority (77%) reported that they were comfortable managing acute pain episodes while 63% expressed comfort with managing chronic pain. Haematologists and SCD specialists showed higher comfort levels prescribing opioids (100% vs 67%, p=0.004) and managing care with hydroxyurea (90% vs 51%, p=0.005) compared with non-haematology providers. Approximately 33% of providers were unaware of the 2014 NHLBI guidelines. Nearly 63% of providers felt patients' medical needs were addressed while only 22% felt their mental health needs were met.

Conclusions: A substantial number of providers did not know about NHLBI's SCD care guidelines. Barriers to providing care for patients with SCD were influenced by providers' specialty, training and practice setting. Increasing provider knowledge could improve hydroxyurea utilisation, pain management and mental health support.
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http://dx.doi.org/10.1136/bmjopen-2021-050880DOI Listing
November 2021

Male-specific late effects in adult hematopoietic cell transplantation recipients: a systematic review from the Late Effects and Quality of Life Working Committee of the Center for International Blood and Marrow Transplant Research and Transplant Complications Working Party of the European Society of Blood and Marrow Transplantation.

Transplant Cell Ther 2021 Oct 29. Epub 2021 Oct 29.

Department of Internal Medicine, Mayo Clinic, Rochester, MN;; Department of Medicine, Sheikh Shakhbout Medical City, Abu Dhabi, UAE.

Background: Male-specific late effects after hematopoietic cell transplantation (HCT) include genital chronic graft-versus-host disease (GvHD), hypogonadism, sexual dysfunction, infertility, and subsequent malignancies, such as prostate, penile, and testicular cancer. They may be closely intertwined and cause prolonged morbidity and decreased quality of life after HCT.

Objective: Here, we provide a systematic review of male-specific late effects in a collaboration between transplant physicians, endocrinologists, urologists, dermatologists, and sexual health professionals through the Late Effects and Quality of Life Working Committee of the Center for International Blood and Marrow Transplant Research, and the Transplant Complications Working Party of the European Society of Blood and Marrow Transplantation.

Study Design: We utilized systematic review methodology to summarize incidence, risk factors, screening, prevention and treatment of these complications and provide consensus evidence-based recommendations for clinical practice and future research.

Results: Most of the evidence regarding male GvHD is still based on limited data, precluding strong therapeutic recommendations. We therefore recommend to systematically screen for male genital GvHD regularly and report it to large registries to allow for a better understanding. Future research should also address treatment since little published evidence is available to date. Male-specific endocrine consequences of HCT include hypogonadism which may also affect bone health. Since the evidence is scarce, current recommendations for hormone substitution and/or bone health treatment are based on similar principles as for the general population. Following HCT, sexual health decreases and this topic should be addressed at regular intervals. Future studies should focus on interventional strategies to address sexual dysfunction. Infertility remains prevalent in patients having undergone myeloablative conditioning, which warrants offering sperm preservation in all HCT candidates. Most studies on fertility rely on descriptive registry analysis and surveys, hence the importance of reporting post-HCT conception data to large registries. Although the quality of evidence is low, the development of cancer in male genital organs does not seem more prevalent than in the general population; however, subsequent malignancies in general seem to be more prevalent in males than females, and special attention should be given to skin and oral mucosa.

Conclusion: Male-specific late effects, probably more under-reported than female-specific complications, should be systematically considered during the regular follow-up visits of male survivors who have undergone HCT. Care of patients with male-specific late effects warrants close collaboration between transplant physicians and specialists from other involved disciplines. Future research should be directed towards better data collection on male-specific late effects and on studies about the interrelationship between these late effects, to allow the development of evidence based effective management practices.
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http://dx.doi.org/10.1016/j.jtct.2021.10.013DOI Listing
October 2021

Digital behavioural interventions for people with sickle cell disease.

Cochrane Libr 2021 27;2021(4). Epub 2021 Apr 27.

Center for Child Health, Behavior and Development, Seattle Children's Research Institute, Seattle, Washington, USA.

This is a protocol for a Cochrane Review (intervention). The objectives are as follows: To identify and assess the effects of digital behavioural interventions focused on behavioural change in people with SCD on: medication adherence or disease management (such as managing acute and chronic pain), or both, on health- and other-related outcomes;specific subgroups defined by age (i.e. children, adolescents and adults) and type of modality or delivery (e.g. cell phone, the Internet).
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http://dx.doi.org/10.1002/14651858.CD014669DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8078570PMC
April 2021

Hydroxyurea Use After Transitions of Care Among Young Adults With Sickle Cell Disease and Tennessee Medicaid Insurance.

JAMA Netw Open 2021 Oct 1;4(10):e2128971. Epub 2021 Oct 1.

Division of Epidemiology, Biostatistics, and Environmental Health, School of Public Health, The University of Memphis, Memphis, Tennessee.

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http://dx.doi.org/10.1001/jamanetworkopen.2021.28971DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8515209PMC
October 2021

Chronic Graft-versus-Host Disease, Nonrelapse Mortality, and Disease Relapse in Older versus Younger Adults Undergoing Matched Allogeneic Peripheral Blood Hematopoietic Cell Transplantation: A Center for International Blood and Marrow Transplant Research Analysis.

Transplant Cell Ther 2021 Oct 9. Epub 2021 Oct 9.

Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin; Fred Hutchinson Cancer Research Center, Seattle, Washington.

The effect of chronic graft-versus-host disease (cGVHD) on the risk of nonrelapse mortality (NRM) and relapse has not been specifically studied in older adults, who are increasingly undergoing allogeneic hematopoietic cell transplantation (alloHCT) and surviving long-term to develop cGVHD. In this Center for International Blood and Marrow Transplant Research (CIBMTR) analysis, we tested our hypothesis that the risk of NRM was higher with the development of cGVHD, particularly among older adults (age ≥60 years). We included 4429 adults age ≥40 years who underwent a first HLA-matched peripheral blood stem cell alloHCT for acute myelogenous leukemia or myelodysplastic syndrome between 2008 and 2017. We compared outcomes of 4 groups-older adults (≥60 years) and younger adults (40 to 59 years) with cGVHD and older and younger adults without cGVHD-to determine the effect of older age and cGVHD on various outcomes. We used Cox proportional hazard models to determine the risk of NRM, relapse, and overall survival (OS). We treated cGVHD as a time-dependent covariate. The severity of cGVHD was based on the CIBMTR clinical definitions. cGVHD was significantly associated with a higher risk of NRM and lower risk of relapse regardless of age. The risk of NRM was higher for older adults versus younger adults. Adults who developed cGVHD as a group had longer OS compared with age-matched cohorts without cGVHD. Older adults had worse OS regardless of cGVHD. Among adults with cGVHD, clinically moderate or severe cGVHD was associated with a significantly higher risk of NRM and lower risk of relapse; severe cGVHD was associated with shorter OS, whereas mild to moderate cGVHD was associated with longer OS. Among both younger and older adults, the development of cGVHD was associated with a higher risk of NRM, lower risk of relapse, and longer OS. Older adults had a higher risk of NRM, but the increased risk of NRM associated with cGVHD did not differ based on age. The development of mild to moderate cGVHD offered the most favorable balance between minimizing NRM and decreasing the risk of relapse. The relapse risk was lowest for adults with severe cGVHD, but high NRM resulted in shorter OS. Developing strategies to avoid clinically severe cGVHD is critically important. © 2021 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc.
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http://dx.doi.org/10.1016/j.jtct.2021.10.002DOI Listing
October 2021

Adherence to immunosuppression in adult heart transplant recipients: A systematic review.

Transplant Rev (Orlando) 2021 12 20;35(4):100651. Epub 2021 Sep 20.

Division of Hematology, Oncology, and Stem Cell Transplant, Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA; Department of Pediatrics, Northwestern University Feinberg School of Medicine, 225 E. Chicago Ave., Chicago, IL 60611, USA. Electronic address:

Background: Successful maintenance of a heart transplant (HTx) graft requires adherence to a triple-drug regimen of immunosuppression. However, achieving adequate adherence can be difficult secondary to complicated dosing regimens, side effects, and mental/emotional barriers. A detailed review of current patterns of adherence to immunosuppression in adult HTx recipients is lacking.

Objective: This systematic review aims to detail the current landscape of adherence to immunosuppression in adult heart transplant patients, including the measurement of adherence, correlates to adherence, health outcomes associated with nonadherence, as well as strategies to improve adherence in HTx patients.

Methods: We conducted searches in PubMed MEDLINE, Embase, CENTRAL register of Controlled Trials (Wiley), and Scopus, from inception to March 2020. Studies were eligible if they outlined an aspect of adherence (as noted above in the objective) to immunosuppression in adult HTx patients. The HTx cohort had to contain at least 10 patients and measurement of adherence had to be done with an objective or otherwise validated measure of adherence (e.g. drug levels, automated pill bottles or adherence questionnaires). Two authors independently screened the articles for inclusion, then subsequently reviewed the full texts of the included articles. Data was extracted into standardized forms and bias evaluations were done using the Newcastle-Ottawa or modified Newcastle-Ottawa tools, depending on the study type. The authors followed all guidelines for the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) statement.

Results: The titles/abstracts of 880 articles were reviewed. Ultimately, 23 articles were included in the final review. The median number of participants was 101, with a range of 19 to 1397. Studies provided information on baseline levels of adherence (17 studies), correlates to adherence (14 studies), health outcomes related to nonadherence (3 studies) and interventions to improve adherence (3 studies). Baseline adherence estimates varied greatly depending on the adherence measure. Multiple significant correlates to nonadherence exist and appear to affect patients with certain sociodemographic backgrounds, those with psychological/psychiatric comorbidities and those with poor support structures. Nonadherence is associated with transplant coronary artery disease and acute late rejection; it may also be associated with long-term mortality. Finally, a simplified dosing regimen with once-a-day tacrolimus as well as use of a mobile phone-based intervention were associated with improved adherence. Bias scores were most deficient due to self-reported outcomes in 18 studies, and lack of controls/adjustments for confounders, in 7 studies.

Conclusions: Adherence to immunosuppression in transplant patients varies, but is associated with observable and modifiable factors which are worth addressing. Further high-quality studies regarding strategies to improve adherence are needed in the literature.
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http://dx.doi.org/10.1016/j.trre.2021.100651DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8527327PMC
December 2021

Post-Transplantation Cyclophosphamide Is Associated with an Increase in Non-Cytomegalovirus Herpesvirus Infections in Patients with Acute Leukemia and Myelodysplastic Syndrome.

Transplant Cell Ther 2021 Sep 26. Epub 2021 Sep 26.

Division of Hematology, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.

The use of post-transplantation cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis in recipients of haploidentical and fully matched transplantations is on the increase. Published studies have reported an increased incidence of cytomegalovirus (CMV) infection with the use of PTCy. Limited data exist on the incidence and outcomes of infection with non-CMV herpesviruses (NCHV) in this setting. The aim of this study was to evaluate the cumulative incidence of NCHV infections and the association of NCHV infections with transplantation-specific outcomes in recipients of haploidentical transplantation with PTCy (HaploCy), matched sibling donor transplantation with PTCy (SibCy), and matched sibling donor transplantation with calcineurin inhibitor-based prophylaxis (SibCNI). We hypothesized that, like CMV infection, HaploCy recipients of also will have a higher risk of NCHV infections. Using the Center for International Blood and Marrow Transplantation Research database, we analyzed 2765 patients (HaploCy, n = 757; SibCNI, n = 1605; SibCy, n = 403) who had undergone their first hematopoietic stem cell transplantation (HCT) between 2012 and 2017 for acute myelogenous leukemia, acute lymphoblastic leukemia, or myelodysplastic syndrome. The cumulative incidence of NCHV at 6 months post-NCT was 13.9% (99% confidence interval], 10.8% to 17.3%) in the HaploCy group, 10.7% (99% CI, 7.1% to 15%) in the SibCy group, and 5.7% (99% CI, 4.3% to 7.3%) in the Sib CNI group (P < .001). This was due primarily to a higher frequency of human herpesvirus 6 viremia reported in patients receiving PTCy. The incidence of Epstein-Barr viremia was low in all groups, and no cases of post-transplantation lymphoproliferative disorder were seen in either PTCy group. The incidence of NCHV organ disease was low in all 3 cohorts. The development of NCHV infection was associated with increased treatment-related mortality, particularly in the HaploCy group. There was no association with the development of GVHD, relapse, or disease-free survival. Patients in PTCy cohorts who did not develop NCHV infection had lower rates of cGVHD. This study demonstrates that the use of PTCy is associated with an increased risk of NCHV infection. The development of NCHV infection was associated with increased nonrelapse mortality, especially in the HaploCy group. Prospective trials should consider viral surveillance strategies in conjunction with assessment of immune reconstitution for a better understanding of the clinical relevance of viral reactivation in different HCT settings.
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http://dx.doi.org/10.1016/j.jtct.2021.09.015DOI Listing
September 2021

A rare case of pancytopenia in a child with cystic fibrosis: Can copper cure it all?

Pediatr Pulmonol 2021 Sep 28. Epub 2021 Sep 28.

Division of Hematology, Oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.

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http://dx.doi.org/10.1002/ppul.25701DOI Listing
September 2021

Risk classification at diagnosis predicts post-HCT outcomes in intermediate-, adverse-risk, and KMT2A-rearranged AML.

Blood Adv 2021 Sep 22. Epub 2021 Sep 22.

University of Texas Southwestern Medical Center, Dallas, Texas, United States.

Little is known about whether risk classification at diagnosis predicts post-hematopoietic cell transplantation (HCT) outcomes for acute myeloid leukemia (AML) patients. We evaluated 8709 AML patients from the CIBMTR database and, after selection and manual curation of cytogenetics data, 3779 patients in CR1 were included in the final analysis: 2384 with intermediate-risk, 969 with adverse-risk, and 426 with KMT2A-rearranged disease. An adjusted multivariable analysis compared to intermediate-risk patients detected an increased risk of relapse for KMT2A-rearranged and adverse-risk patients (HR 1.27, p = 0.01 and HR 1.71, p < 0.001, respectively). Leukemia-free survival (LFS) was similar for KMT2A and adverse-risk patients (HR 1.26, p = 0.002 and HR 1.47, p < 0.001), as was overall survival (OS) (HR 1.32, p < 0.001 and HR 1.45, p < 0.001). No differences in outcome could be detected when patients were stratified by KMT2A fusion partner. This is the largest study conducted to date on post-HCT outcomes in AML using manually curated cytogenetics for risk stratification. Our work demonstrates that risk classification at diagnosis remains predictive of post-HCT outcomes in AML. It also highlights the critical need to develop novel treatment strategies for patients with KMT2A rearrangements and adverse-risk disease.
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http://dx.doi.org/10.1182/bloodadvances.2021004881DOI Listing
September 2021

Haploidentical vs. sibling, unrelated, or cord blood hematopoietic cell transplantation for acute lymphoblastic leukemia.

Blood Adv 2021 Sep 21. Epub 2021 Sep 21.

University of Virginia, Charlottesville, Virginia, United States.

The role of haploidentical hematopoietic cell transplantation (HCT) using post-transplant cyclophosphamide (PTCy) for acute lymphoblastic leukemia (ALL) is being defined. We performed a retrospective, multivariate analysis comparing outcomes of HCT approaches by donor for adults with ALL in remission. The primary objective was to compare overall survival (OS) between haploidentical HCT using PTCy and HLA-matched sibling donor (MSD), 8/8 HLA-matched unrelated donor (MUD) , 7/8 HLA-matched UD, or umbilical cord blood (UCB) HCT. Comparing haploidentical to MSD HCT, OS, leukemia-free survival (LFS), non-relapse mortality (NRM), relapse, and acute graft-versus-host disease (aGVHD) were not different but chronic GVHD (cGVHD) was higher with MSD HCT. Compared to MUD HCT, OS, LFS, and relapse were not different but MUD HCT had increased NRM (HR 1.42, P=0.02), grade 3-4 aGVHD (HR 1.59, P=0.005), and cGVHD. Compared to 7/8 UD HCT, LFS and relapse were not different, but 7/8 UD HCT had worse OS (HR 1.38, P=0.01) and increased NRM (HR 2.13, P=<0.001), grade 3-4 aGVHD (HR 1.86, P=0.003), and cGVHD (HR 1.72, P=<0.001). Compared to UCB HCT, late OS , late LFS, relapse, and cGVHD were not different but UCB HCT had worse early OS (≤18 months, HR 1.93, P<0.001), worse early LFS (HR 1.40, P=0.007) and increased incidences of NRM (HR 2.08, P<0.001) and grade 3-4 aGVHD (HR 1.97, P<0.001). Haploidentical HCT using PTCy showed no difference in survival but less GVHD compared to traditional MSD and MUD HCT and is the preferred alternative donor HCT option for adults with ALL in CR.
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http://dx.doi.org/10.1182/bloodadvances.2021004916DOI Listing
September 2021

Recurrent thrombosis with a mispositioned stent after treatment of an adolescent with May-Thurner syndrome.

Pediatr Blood Cancer 2021 Sep 14:e29350. Epub 2021 Sep 14.

Department of Pediatrics, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.

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http://dx.doi.org/10.1002/pbc.29350DOI Listing
September 2021

Pancytopenia in a child with cystic fibrosis and severe copper deficiency: Insight from bone marrow evaluation.

Pediatr Blood Cancer 2021 Dec 31;68(12):e29276. Epub 2021 Jul 31.

Division of Hematology, Oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.

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http://dx.doi.org/10.1002/pbc.29276DOI Listing
December 2021

Telemedicine in Malignant and Nonmalignant Hematology: Systematic Review of Pediatric and Adult Studies.

JMIR Mhealth Uhealth 2021 07 8;9(7):e29619. Epub 2021 Jul 8.

Division of Pediatric Hematology, Oncology, Neuro-Oncology & Stem Cell Transplantation, Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL, United States.

Background: Telemedicine, including video-, web-, and telephone-based interventions, is used in adult and pediatric populations to deliver health care and communicate with patients. In the realm of hematology, telemedicine has recently been used to safely and efficiently monitor treatment side-effects, perform consultations, and broaden the reach of subspecialty care.

Objective: We aimed to synthesize and analyze information regarding the feasibility, acceptability, and potential benefits of telemedicine interventions in malignant and nonmalignant hematology, as well as assess the recognized limitations of these interventions.

Methods: Studies were identified through a comprehensive Medical Subject Headings (MeSH) search on the PubMed MEDLINE, Controlled Register of Clinical Trials (Cochrane CENTRAL from Wiley), Embase, and CINAHL (EBSCO) databases on February 7, 2018. A second search, utilizing the same search strategy, was performed on October 1, 2020. We followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines in the reporting of included evidence. Included studies were original articles researching the feasibility, acceptability, and clinical outcomes of telemedicine or telehealth interventions in pediatric or adult populations with malignant or nonmalignant hematological conditions. Data items in the extraction form included first author name, publication year, country, malignant or nonmalignant hematological condition or disease focus of the study, participant age, participant age subgroup (pediatric or adult), study design and setting, telemedicine intervention type and description, study purpose, and main study outcomes.

Results: A total of 32 articles met the preset criteria and were included in this study. Most (25/32) studies were conducted in adults, and the remaining (7/32) were conducted in the pediatric population. Of the 32 studies, 12 studied malignant hematological conditions, 18 studied nonmalignant conditions, and two studied both malignant and nonmalignant conditions. Study types included pilot study (11/32), retrospective study (9/32), randomized controlled trial (6/32), cross-sectional study (2/32), case study (1/32), pre-post study (1/32), noncomparative prospective study (1/32), and prospective cohort study (1/32). The three main types of telemedicine interventions utilized across all studies were video-based (9/32), telephone-based (9/32), and web-based interventions (14/32). Study results showed comparable outcomes between telemedicine and traditional patient encounter groups across both pediatric and adult populations for malignant and nonmalignant hematological conditions.

Conclusions: Evidence from this review suggests that telemedicine use in nonmalignant and malignant hematology provides similar or improved health care compared to face-to-face encounters in both pediatric and adult populations. Telemedicine interventions utilized in the included studies were well received in both pediatric and adult settings. However, more research is needed to determine the efficacy of implementing more widespread use of telemedicine for hematological conditions.
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http://dx.doi.org/10.2196/29619DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8299344PMC
July 2021

Adherence to Immunosuppression Medications among Heart Transplant Recipients: Challenges, Opportunities, and Potential Role of Digital Approaches in the COVID-19 Era.

J Cardiovasc Dev Dis 2021 Jun 10;8(6). Epub 2021 Jun 10.

Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA.

Society and medical practice have been restructured dramatically to avoid further spread of the COVID-19 virus; telehealth/telemedicine, mask wearing, and nationwide social distancing practices have become widespread. However, we still face unprecedented challenges in fields where patients require frequent and active follow-up visits for monitoring, including that of solid-organ transplant, and in particular, heart transplant. Adherence to immunosuppression remains a unique challenge in heart transplantation, especially during the COVID-19 pandemic. Failure to adhere to immunosuppression can have disastrous consequences, including graft rejection and death. In this article, we discuss challenges related to adherence to immunosuppression medications among heart transplant recipients, as well as opportunities to leverage digital approaches and interventions to monitor and optimize adherence behavior and health outcomes in this population.
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http://dx.doi.org/10.3390/jcdd8060068DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8230436PMC
June 2021

The ASH-ASPHO Choosing Wisely Campaign: 5 hematologic tests and treatments to question.

Pediatr Blood Cancer 2021 Aug 28;68(8):e28967. Epub 2021 May 28.

Division of Hematology/Oncology, St. Michael's Hospital, Toronto, ON, Canada.

Choosing Wisely is a medical stewardship and quality-improvement initiative led by the American Board of Internal Medicine Foundation in collaboration with leading medical societies in the United States. The American Society of Hematology (ASH) has been an active participant in the Choosing Wisely project. In 2019, ASH and the American Society of Pediatric Hematology/Oncology (ASPHO) formed a joint task force to solicit, evaluate, and select items for a pediatric-focused Choosing Wisely list. By using an iterative process and an evidence-based method, the ASH-ASPHO Task Force identified 5 hematologic tests and treatments that health care providers and patients should question because they are not supported by evidence, and/or they involve risks of medical and financial costs with low likelihood of benefit. The ASH-ASPHO Choosing Wisely recommendations are as follows: (1) avoid routine preoperative hemostatic testing in an otherwise healthy child with no previous personal or family history of bleeding, (2) avoid platelet transfusion in asymptomatic children with a platelet count 10 × 10 /μL unless an invasive procedure is planned, (3) avoid thrombophilia testing in children with venous access-associated thrombosis and no positive family history, (4) avoid packed red blood cells transfusion for asymptomatic children with iron deficiency anemia and no active bleeding, and (5) avoid routine administration of granulocyte colony-stimulating factor for prophylaxis of children with asymptomatic autoimmune neutropenia and no history of recurrent or severe infections. We recommend that health care providers carefully consider the anticipated risks and benefits of these identified tests and treatments before performing them.
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http://dx.doi.org/10.1002/pbc.28967DOI Listing
August 2021

Fertility preservation education for pediatric hematology-oncology fellows, faculty and advanced practice providers: a pilot study.

Pediatr Hematol Oncol 2021 May 24:1-6. Epub 2021 May 24.

Division of Hematology, Oncology, Neuro-Oncology & Stem Cell Transplantation, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.

Infertility secondary to chemotherapy, myeloablative conditioning regimens prior to stem cell transplantation, radiation therapy, and/or surgery is an important cause of morbidity and psychosocial distress among pediatric cancer patients. Known options exist for fertility preservation; however, knowledge among providers varies. We conducted a pilot study with an educational intervention over one-hour for hematology-oncology faculty, fellows, and advanced practice providers. Participants completed pre-/post-test assessment on fertility preservation knowledge. Participants' pretest mean (SD) score was 53% (17%), which significantly increased to 72% (11%) in the post-test ( = 0.0004). We demonstrated that a fertility education intervention could improve knowledge regarding infertility risk assessment and fertility preservation options.
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http://dx.doi.org/10.1080/08880018.2021.1928348DOI Listing
May 2021

Engaging Caregivers and Providers of Children With Sickle Cell Anemia in Shared Decision Making for Hydroxyurea: Protocol for a Multicenter Randomized Controlled Trial.

JMIR Res Protoc 2021 May 21;10(5):e27650. Epub 2021 May 21.

Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, United States.

Background: Sickle cell anemia (SCA) is a genetic blood disorder that puts children at a risk of serious medical complications, early morbidity and mortality, and high health care utilization. Until recently, hydroxyurea was the only disease-modifying treatment for this life-threatening disease and has remained the only option for children younger than 5 years. Evidence-based guidelines recommend using a shared decision-making (SDM) approach for offering hydroxyurea to children with SCA (HbSS or HbS/β0 thalassemia) aged as early as 9 months. However, the uptake remains suboptimal, likely because caregivers lack information about hydroxyurea and have concerns about its safety and potential long-term side effects. Moreover, clinicians do not routinely receive training or tools, especially those that provide medical evidence and consider caregivers' preferences and values, to facilitate a shared discussion with caregivers.

Objective: The aim of this study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that considers medical evidence and parent values and preferences).

Methods: We designed our study to compare the effectiveness of two methods for disseminating hydroxyurea guidelines to facilitate SDM: a clinician pocket guide (ie, usual care) and a clinician hydroxyurea SDM toolkit (H-SDM toolkit). Our primary outcomes are caregiver reports of decisional uncertainty and knowledge of hydroxyurea. The study also assesses the number of children (aged 0-5 years) who were offered and prescribed hydroxyurea and the resultant health outcomes.

Results: The Ethics Committee of the Cincinnati Children's Hospital Medical Center approved this study in November 2017. As of February 2021, we have enrolled 120 caregiver participants.

Conclusions: The long-term objective of this study is to improve the quality of care for children with SCA. Using multicomponent dissemination methods developed in partnership with key stakeholders and designed to address barriers to high-quality care, caregivers of patients with SCA can make informed and shared decisions about their health.

Trial Registration: ClinicalTrials.gov NCT03442114; https://clinicaltrials.gov/ct2/show/NCT03442114.

International Registered Report Identifier (irrid): DERR1-10.2196/27650.
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http://dx.doi.org/10.2196/27650DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8178738PMC
May 2021

Comparing the Effectiveness of Education Versus Digital Cognitive Behavioral Therapy for Adults With Sickle Cell Disease: Protocol for the Cognitive Behavioral Therapy and Real-time Pain Management Intervention for Sickle Cell via Mobile Applications (CaRISMA) Study.

JMIR Res Protoc 2021 May 14;10(5):e29014. Epub 2021 May 14.

Department of Medicine, University of Pittsburgh, Pittsburgh, PA, United States.

Background: Patients with sickle cell disease (SCD) experience significant medical and psychological stressors that affect their mental health, well-being, and disease outcomes. Digital cognitive behavioral therapy (CBT) has been used in other patient populations and has demonstrated clinical benefits. Although evidence-based, nonpharmacological interventions for pain management are widely used in other populations, these treatments have not been well studied in SCD. Currently, there are no adequately powered large-scale clinical trials to evaluate the effectiveness and dissemination potential of behavioral pain management for adults with SCD. Furthermore, some important details regarding behavioral therapies in SCD remain unclear-in particular, what works best for whom and when.

Objective: Our primary goal is to compare the effectiveness of two smartphone-delivered programs for reducing SCD pain symptoms: digital CBT versus pain and SCD education (Education). Our secondary goal is to assess whether baseline depression symptoms moderate the effect of interventions on pain outcomes. We hypothesize that digital CBT will confer greater benefits on pain outcomes and depressive symptoms at 6 months and a greater reduction in health care use (eg, opioid prescriptions or refills or acute care visits) over 12 months.

Methods: The CaRISMA (Cognitive Behavioral Therapy and Real-time Pain Management Intervention for Sickle Cell via Mobile Applications) study is a multisite comparative effectiveness trial funded by the Patient-Centered Outcomes Research Institute. CaRISMA is conducted at six clinical academic sites, in partnership with four community-based organizations. CaRISMA will evaluate the effectiveness of two 12-week health coach-supported digital health programs with a total of 350 participants in two groups: CBT (n=175) and Education (n=175). Participants will complete a series of questionnaires at baseline and at 3, 6, and 12 months. The primary outcome will be the change in pain interference between the study arms. We will also evaluate changes in pain intensity, depressive symptoms, other patient-reported outcomes, and health care use as secondary outcomes. We have 80% power to detect a difference of 0.37 SDs between study arms on 6-month changes in the outcomes with 15% expected attrition at 6 months. An exploratory analysis will examine whether baseline depression symptoms moderate the effect of the intervention on pain interference.

Results: This study will be conducted from March 2021 through February 2022, with results expected to be available in February 2023.

Conclusions: Patients with SCD experience significant disease burden, psychosocial stress, and impairment of their quality of life. CaRISMA proposes to leverage digital technology and overcome barriers to the routine use of behavioral treatments for pain and depressive symptoms in the treatment of adults with SCD. The study will provide data on the comparative effectiveness of digital CBT and Education approaches and evaluate the potential for implementing evidence-based behavioral interventions to manage SCD pain.

Trial Registration: ClinicalTrials.gov NCT04419168; https://clinicaltrials.gov/ct2/show/NCT04419168.

International Registered Report Identifier (irrid): PRR1-10.2196/29014.
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http://dx.doi.org/10.2196/29014DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8164118PMC
May 2021

Systematic Reviews in Hematopoietic Cell Transplantation and Cellular Therapy: Considerations and Guidance from the American Society for Transplantation and Cellular Therapy, European Society for Blood and Marrow Transplantation, and Center for International Blood and Marrow Transplant Research Late Effects and Quality of Life Working Committee.

Transplant Cell Ther 2021 05 28;27(5):380-388. Epub 2021 Jan 28.

Center for International Blood and Marrow Transplant Research, Milwaukee, Wisconsin. Electronic address:

Systematic reviews apply rigorous methodologies to address a prespecified, clearly formulated clinical research question. The conclusion that results is often cited to more robustly inform decision making by clinicians, third-party payers, and managed care organizations about the clinical question of interest. Although systematic reviews provide a rigorous standard, they may be infeasible when the task is to create general disease-focused guidelines comprising multiple clinical practice questions versus a single major clinical practice question. Collaborating transplantation and cellular therapy society committees also recognize that the quantity and or quality of reference sources may be insufficient for a meaningful systematic review. As the conduct of systematic reviews has evolved over time in terms of grading systems, reporting requirements, and use of technology, here we provide current guidance on methodologies, resources for reviewers, and approaches to overcome challenges in conducting systematic reviews in transplantation and cellular therapy.
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http://dx.doi.org/10.1016/j.jtct.2020.12.002DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8415092PMC
May 2021

Return to Work Among Young Adult Survivors of Allogeneic Hematopoietic Cell Transplantation in the United States.

Transplant Cell Ther 2021 08 22;27(8):679.e1-679.e8. Epub 2021 Apr 22.

Center for International Blood and Marrow Transplant Research), National Marrow Donor Program/Be The Match, Minneapolis, Minnesota.

Young adult (YA) survivors of allogeneic hematopoietic cell transplantation (HCT) are at risk for late psychosocial challenges, including the inability to return to work post-HCT. Work-related outcomes in this population remain understudied, however. We conducted this study to assess the post-HCT work status of survivors of allogeneic HCT who underwent HCT as YAs and to analyze the patient-, disease-, and HCT-related factors associated with their work status at 1 year post-HCT. Using Center for International Blood and Marrow Transplant Research data, we evaluated the post-HCT work status (full-time, part-time work, unemployed, or medical disability) of 1365 YA HCT survivors who underwent HCT between 2008 and 2015. Percentages of work status categories were reported at 4 time points: 6 months, 1 year, 2 years, and 3 years post-HCT. Percentages of post-HCT work status categories at the 1-year time point were also described in relation to survivors' pre-HCT work status categories. Factors associated with 1-year post-HCT work status (full-time or part-time work) were examined using logistic regression. From 6 months to 3 years post-HCT, the percentage of survivors working full-time increased from 18.3% to 50.7% and the percentage working part-time increased from 6.9% to 10.5%. Of patients in full-time work pre-HCT, 50% were unemployed or on medical disability at 1 year post-HCT. Female sex (odds ratio [OR], 0.55; 95% confidence interval [CI], 0.40 to 0.77), HCT Comorbidity Index score ≥3 (OR, 0.57; 95% CI, 0.39 to 0.82), pre-HCT unemployment (OR, 0.37; 95% CI, 0.24 to 0.56), medical disability (OR, 0.44; 95% CI, 0.28 to 0.70), development of grade III-IV acute graft-versus-host disease (OR, 0.52; 95% CI, 0.34 to 0.80), and relapse within 1 year post-HCT (OR, 0.34; 95% CI, 0.21 to 0.56) were associated with a lower likelihood of employment at 1 year post-HCT. Compared with myeloablative conditioning (MAC) with total body irradiation (TBI), MAC without TBI (OR, 1.71; 95% CI, 1.16 to 2.53) was associated with a greater likelihood of employment at 1 year post-HCT. Graduate school-level education (OR, 2.47; 95% CI, 1.49 to 4.10) was also associated with a greater likelihood of employment at 1 year post-HCT. Although the work status among YA HCT survivors continued to improve over time, a substantial subset became or remained unemployed or on medical disability. These findings underscore the need for effective interventions to support return to work in this population.
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http://dx.doi.org/10.1016/j.jtct.2021.04.013DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8425287PMC
August 2021

Genetics of HLA Peptide Presentation and Impact on Outcomes in HLA-Matched Allogeneic Hematopoietic Cell Transplantation.

Transplant Cell Ther 2021 07 18;27(7):591-599. Epub 2021 Apr 18.

Internal Medicine, University of North Carolina, Chapel Hill, North Carolina; BMTCT Program, Division of Hematology and Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, North Carolina. Electronic address:

Minor histocompatibility antigens (mHAs), recipient-derived peptide epitopes presented on the cell surface, are known to mediate graft-versus-host disease (GVHD); however, there are no current methods to associate mHA features with GVHD risk. This deficiency is due in part to the lack of technological means to accurately predict, let alone confirm, the tremendous number of potential mHAs in each individual transplant. Previous studies have shown that different HLA molecules present varying fractions of candidate peptide epitopes; however, the genetic "distance" between HLA-matched donors and recipients is relatively constrained. From these 2 observations, it is possible that the HLA type for a donor-recipient pair (DRP) would provide a surrogate measurement of the number of predicted mHAs, which could be related to GVHD risk. Because different HLA molecules present variable numbers of peptide antigens, a predicted cumulative peptide-binding efficiency can be calculated for individual DRP based on the pair's HLA type. The purpose of this study was to test whether cumulative peptide-binding efficiency is associated with the risk of acute GVHD (aGVHD) or relapse. In this retrospective Center for International Blood and Marrow Transplant Research study, a total of 3242 HLA-matched DRPs were analyzed for predicted cumulative peptide-binding efficiency using their HLA types and were divided into tertiles based on their scores. Univariable and multivariable analyses was performed to test for associations between cumulative peptide-binding efficiency for DRPs, divided into the HLA-matched related donor (MRD) and HLA-matched unrelated donor (MUD) cohorts, and the primary outcomes of aGVHD and relapse. Secondary outcomes investigated included overall survival, disease-free survival, and transplantation-related mortality. Using a computationally generated peptidome as a test dataset, the tested series of HLA class I displayed peptide-binding frequencies ranging from 0.1% to 3.8% of the full peptidome, and HLA class II molecules had peptide-binding frequencies of 12% to 77% across the HLA-DRB1 allotypes. By increasing binding efficiency tertile, the cumulative incidence of aGVHD at 6 months for MUD patients was 41%, 41%, and 45% for HLA class I (P = .336) and 44%, 41%, and 42% for HLA class II (P = .452). The cumulative incidences of relapse at 3 years for MUD transplant recipients were 36%, 38%, and 38% for HLA class I (P = .533) and 37%, 37%, and 38% for HLA class II (P = .896). The findings were similar for MRD transplant recipients. Multivariable analysis did not identify any impact of peptide-binding efficiency on aGVHD or relapse in MUD or MRD transplant recipients. Whereas GVHD is mediated by minor antigen mismatches in the context of HLA-matched allo-HCT, peptide-binding efficiency, which was used as a surrogate measurement for predicted number of binding antigens, did not provide additional clinical information for GVHD risk assessment. The negative result may be due to the limitations of this surrogate marker, or it is possible that GVHD is driven by a subset of immunogenic mHAs. Further research should be directed at direct mHA epitope and immunogenicity prediction.
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http://dx.doi.org/10.1016/j.jtct.2021.04.003DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8343993PMC
July 2021

Scurvy and non-immune hemolytic anemia in an adolescent with trisomy 21.

Pediatr Blood Cancer 2021 Aug 21;68(8):e29070. Epub 2021 Apr 21.

Division of Hematology, Oncology, and Stem Cell Transplantation, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.

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http://dx.doi.org/10.1002/pbc.29070DOI Listing
August 2021

Impact of depth of clinical response on outcomes of acute myeloid leukemia patients in first complete remission who undergo allogeneic hematopoietic cell transplantation.

Bone Marrow Transplant 2021 09 16;56(9):2108-2117. Epub 2021 Apr 16.

Haematology Research Centre, Department of Immunology and Inflammation, Imperial College London, London, UK.

Acute myeloid leukemia (AML) patients often undergo allogeneic hematopoietic cell transplantation (alloHCT) in first complete remission (CR). We examined the effect of depth of clinical response, including incomplete count recovery (CRi) and/or measurable residual disease (MRD), in patients from the Center for International Blood and Marrow Transplantation Research (CIBMTR) registry. We identified 2492 adult patients (1799 CR and 693 CRi) who underwent alloHCT between January 1, 2007 and December 31, 2015. The primary outcome was overall survival (OS). Multivariable analysis was performed to adjust for patient-, disease-, and transplant-related factors. Baseline characteristics were similar. Patients in CRi compared to those in CR had an increased likelihood of death (HR: 1.27; 95% confidence interval: 1.13-1.43). Compared to CR, CRi was significantly associated with increased non-relapse mortality (NRM), shorter disease-free survival (DFS), and a trend toward increased relapse. Detectable MRD was associated with shorter OS, shorter DFS, higher NRM, and increased relapse compared to absence of MRD. The deleterious effects of CRi and MRD were independent. In this large CIBMTR cohort, survival outcomes differ among AML patients based on depth of CR and presence of MRD at the time of alloHCT. Further studies should focus on optimizing post-alloHCT outcomes for patients with responses less than CR.
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http://dx.doi.org/10.1038/s41409-021-01261-6DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8425595PMC
September 2021

Broad-Spectrum Antibiotics and Risk of Graft-versus-Host Disease in Pediatric Patients Undergoing Transplantation for Acute Leukemia: Association of Carbapenem Use with the Risk of Acute Graft-versus-Host Disease

Transplant Cell Ther 2021 02 21;27(2):177.e1-177.e8. Epub 2020 Dec 21.

Center for Pediatric Clinical Effectiveness, Children's Hospital of Philadelphia, Philadelphia, PA.

Variation in the gastrointestinal (GI) microbiota after hematopoietic cell transplantation (HCT) has been associated with acute graft-versus-host disease (aGVHD). Because antibiotics induce dysbiosis, we examined the association of broad-spectrum antibiotics with subsequent aGVHD risk in pediatric patients undergoing HCT for acute leukemia. We performed a retrospective analysis in a dataset merged from 2 sources: (1) the Center for International Blood and Marrow Transplant Research, an observational transplantation registry, and (2) the Pediatric Health Information Services, an administrative database from freestanding children's hospitals. We captured exposure to 3 classes of antibiotics used for empiric treatment of febrile neutropenia: (1) broad-spectrum cephalosporins, (2) antipseudomonal penicillins, and (3) carbapenems. The primary outcome was grade II-IV aGVHD; secondary outcomes were grade III-IV aGVHD and lower GI GVHD. The adjusted logistic regression model (full cohort) and time-to-event analysis (subcohort) included transplantation characteristics, GVHD risk factors, and adjunctive antibiotic exposures as covariates. The full cohort included 2550 patients at 36 centers; the subcohort included 1174 patients. In adjusted models, carbapenems were associated with an increased risk of grade II-IV aGVHD in the full cohort (adjusted odds ratio [aOR], 1.24; 95% confidence interval [CI], 1.02 to 1.51) and subcohort (sub hazard ratio [HR], 1.31; 95% CI, 0.99 to 1.72), as well as with an increased risk of grade III-IV aGVHD (subHR, 1.77; 95% CI, 1.25 to 2.52). Early carbapenem exposure (before day 0) especially impacted aGVHD risk. For antipseudomonal penicillins, the associations with aGVHD were in the direction of increased risk but were not statistically significant. There was no identified association between broad-spectrum cephalosporins and aGVHD. Carbapenems, more than other broad-spectrum antibiotics, should be used judiciously in pediatric HCT recipients to minimize aGVHD risk. Further research is needed to clarify the mechanism underlying this association.
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http://dx.doi.org/10.1016/j.jtct.2020.10.012DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7946150PMC
February 2021

Access to Technology and Preferences for an mHealth Intervention to Promote Medication Adherence in Pediatric Acute Lymphoblastic Leukemia: Approach Leveraging Behavior Change Techniques.

J Med Internet Res 2021 02 18;23(2):e24893. Epub 2021 Feb 18.

Division of Hematology, Oncology and Stem Cell Transplantation, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, United States.

Background: Suboptimal adherence to 6-mercaptopurine (6-MP) is prevalent in pediatric acute lymphoblastic leukemia (ALL) and associated with increased risk of relapse. Rapid uptake of personal technology makes mobile health (mHealth) an attractive platform to promote adherence.

Objective: Study objectives were to examine access to mobile technology and preferences for an mHealth intervention to improve medication adherence in pediatric ALL.

Methods: A cross-sectional survey was administered in oncology clinic to parents of children with ALL as well as adolescents and young adults (AYAs) with ALL receiving maintenance chemotherapy.

Results: A total of 49 parents (median age [IQR] 39 [33-42] years; female 76% [37/49]) and 15 patients (median age [IQR] 17 [16-19]; male 80% [12/15]) participated. All parents and AYAs owned electronic tablets, smartphones, or both. Parents' most endorsed mHealth app features included a list of medications (71%, 35/49), information about 6-MP (71%, 35/49), refill reminders (71%, 35/49), and reminders to take 6-MP (71%, 35/49). AYAs' most endorsed features included refill reminders (73%, 11/15), reminders to take 6-MP (73%, 11/15), and tracking 6-MP (73%, 11/15).

Conclusions: Parents and AYAs reported ubiquitous access to mobile technology and strong interest in multiple adherence-specific mHealth app features. Parents and AYAs provided valuable insight into preferred features for a multifunctional behavioral intervention (mHealth app) to promote medication adherence in pediatric ALL.
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http://dx.doi.org/10.2196/24893DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7932843PMC
February 2021
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