Publications by authors named "Robert I Liem"

61 Publications

Heart rate variability associated with acute exercise challenge in children with sickle cell anaemia.

Br J Haematol 2021 Sep 22. Epub 2021 Sep 22.

Division of Hematology, Oncology, & Stem Cell Transplant, Department of Pediatrics, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, USA.

We examined heart rate variability (HRV) during exercise testing in 20 children with sickle cell anaemia (SCA) and 12 controls. Subjects achieved lower median HRV at peak exercise [standard deviation of R-wave to R-wave intervals (SDNN), 2·3 vs 2·9 ms, P = 0·027; logarithmic transformation of high frequency power (lnHF), 0·9 vs 1·3 ln(ms ), P = 0·047] and had lower post-exercise HRV across minute-by-minute analysis of recovery. After adjustment for haemoglobin, fitness and SCA status, subjects had lower HRV at the end of recovery with differences increasing as baseline HRV increased. Further investigation of HRV and exercise safety in SCA is warranted.
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http://dx.doi.org/10.1111/bjh.17834DOI Listing
September 2021

Cerebral and skeletal muscle tissue oxygenation during exercise challenge in children and young adults with sickle cell anaemia.

Br J Haematol 2021 Jul 26. Epub 2021 Jul 26.

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

We used near-infrared spectroscopy to examine tissue oxygenation (StO during exercise in 17 children and young adults with sickle cell anaemia (SCA) and 13 controls. Patients had lower cerebral StO at all exercise stages and demonstrated significantly greater decreases in cerebral StO later during exercise. Quadriceps StO increased similarly in patients and controls during early exercise, but decreases from baseline were greater in patients during later exercise. At similar workloads, patients demonstrated lower cerebral StO (69·2 ± 6·6 vs. 79·5 ± 5·3%, P < 0·001) and trended towards lower quadriceps StO (67·7 ± 9·0 vs. 73·2 ± 7·9%, P = 0·09) Further studies of tissue oxygenation during exercise in SCA are warranted.
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http://dx.doi.org/10.1111/bjh.17724DOI Listing
July 2021

Safety of maximal cardiopulmonary exercise testing in individuals with sickle cell disease: a systematic review.

Br J Sports Med 2021 Jul 20. Epub 2021 Jul 20.

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

Objective: We evaluated the safety of maximal cardiopulmonary exercise testing (CPET) in individuals with sickle cell disease (SCD). Maximal CPET using gas exchange analysis is the gold standard for measuring cardiopulmonary fitness in the laboratory, yet its safety in the SCD population is unclear.

Design: Systematic review.

Data Sources: Systematic search of Medline (PubMed), EMBASE, Cochrane, ClinicalTrials.gov and professional society websites for all published studies and abstracts through December 2020.

Eligibility Criteria For Selecting Studies: Two reviewers independently extracted data of interest from studies that assessed safety outcomes of maximal CPET in children and adults with SCD. A modified version of the Newcastle-Ottawa Scale was used to assess for risk of bias in studies included.

Results: In total, 24 studies met inclusion/exclusion criteria. Adverse events were reported separately or as part of study results in 36 (3.8%) of 939 participants with SCD undergoing maximal CPET in studies included. Most adverse events were related to transient ischaemic changes on ECG monitoring or oxygen desaturation during testing, which did not result in arrhythmias or other complications. Only 4 (0.43%) of 939 participants experienced pain events due to maximal CPET.

Conclusion: Maximal CPET appears to be a safe testing modality in children and adults with SCD and can be used to better understand the physiological basis of reduced exercise capacity and guide exercise prescription in this population. Some studies did not focus on reporting adverse events related to exercise testing or failed to mention safety monitoring, which contributed to risk of bias.
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http://dx.doi.org/10.1136/bjsports-2021-104450DOI Listing
July 2021

A case of severe bi-cytopenias and hypocellular bone marrow with uncontrolled nephropathic cystinosis.

Pediatr Blood Cancer 2021 Jun 8;68(6):e28952. Epub 2021 Feb 8.

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.28952DOI Listing
June 2021

A systematic review of quality of life in sickle cell disease and thalassemia after stem cell transplant or gene therapy.

Blood Adv 2021 01;5(2):570-583

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

Patients with sickle cell disease (SCD) and thalassemia experience several complications across their lifespan that lead to impairment in different health-related quality of life (HRQOL) domains. There is increasing interest in curative therapies for patients with SCD and thalassemia, including hematopoietic stem cell transplant (HSCT) and gene therapy; however, the effect of these therapies on various HRQOL domains remains unclear. Our objective was to systematically evaluate the most recent evidence for the effect of HSCT and gene therapy on HRQOL in patients with SCD and thalassemia. A systematic search of medical literature databases was conducted. A total of 16 studies (thalassemia, n = 9; SCD, n = 6; both, n = 1) involving 517 participants met inclusion criteria (thalassemia, n = 416; SCD, n = 101). HSCT was associated with a small to large positive effects in most HRQOL domains (Cohen's d; mean = 0.47; median = 0.37; range, 0.27-2.05). In thalassemia, HSCT was frequently associated with large positive effects in physical and emotional HRQOL domains (median d = 0.79 and d = 0.57, respectively). In SCD, HSCT was associated with large positive effects in all HRQOL domains. Emerging data suggest improvement in HRQOL outcomes across different domains following gene therapy in thalassemia and SCD. The quality of evidence was moderate in 13 studies (81%). HSCT has a positive impact on several HRQOL domains in patients with SCD and thalassemia; however, more longitudinal studies are warranted to assess the sustainability of these effects. Reporting HRQOL outcomes from ongoing gene therapy or gene-editing trials in SCD and thalassemia is key to better understand the benefits of such therapies.
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http://dx.doi.org/10.1182/bloodadvances.2020002948DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7839355PMC
January 2021

Impact of Medicaid expansion on access and healthcare among individuals with sickle cell disease.

Pediatr Blood Cancer 2020 05 8;67(5):e28152. Epub 2020 Mar 8.

Department of Hematology/Oncology, University of California San Francisco Benioff Children's Hospital, Oakland, Illinois.

Purpose: Sickle cell disease (SCD) is associated with high acute healthcare utilization. The purpose of this study was to examine whether Medicaid expansion in California increased Medicaid enrollment, increased hydroxyurea prescriptions filled, and decreased acute healthcare utilization in SCD.

Methods: Individuals with SCD (≤65 years and enrolled in Medicaid for ≥6 total calendar months any year between 2011 and 2016) were identified in a multisource database maintained by the California Sickle Cell Data Collection Program. We describe trends and changes in Medicaid enrollment, hydroxyurea prescriptions filled, and emergency department (ED) visits and hospital admissions before (2011-2013) and after (2014-2016) Medicaid expansion in California.

Results: The cohort included 3635 individuals. Enrollment was highest in 2014 and lowest in 2016 with a 2.8% annual decease postexpansion. Although <20% of the cohort had a hydroxyurea prescription filled, the percentage increased by 5.2% annually after 2014. The ED visit rate was highest in 2014 and decreased slightly in 2016, decreasing by 1.1% annually postexpansion. Hospital admission rates were similar during the pre- and postexpansion periods. Young adults and adults had higher ED and hospital admission rates than children and adolescents.

Conclusions: Medicaid expansion does not appear to have improved enrollment or acute healthcare utilization among individuals with SCD in California. Future studies should explore whether individuals with SCD transitioned to other insurance plans or became uninsured postexpansion, the underlying reasons for low hydroxyurea utilization, and the lack of effect on hospital admissions despite a modest effect on ED visits.
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http://dx.doi.org/10.1002/pbc.28152DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7096276PMC
May 2020

Translating exercise benefits in sickle cell disease.

Blood 2019 12;134(25):2227-2229

Ann & Robert H. Lurie Children's Hospital of Chicago.

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http://dx.doi.org/10.1182/blood.2019003651DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8212353PMC
December 2019

2019 sickle cell disease guidelines by the American Society of Hematology: methodology, challenges, and innovations.

Blood Adv 2019 12;3(23):3945-3950

Department of Medicine, University of Kansas Medical Center, Kansas City, KS.

The American Society of Hematology (ASH) convened 5 guideline panels to develop clinical practice recommendations addressing 5 management areas of highest importance to individuals living with sickle cell disease: pain, cerebrovascular complications, pulmonary and kidney complications, transfusion, and hematopoietic stem cell transplant. Panels were multidisciplinary and consisted of patient representatives, content experts, and methodologists. The Mayo Clinic Evidence-Based Practice Center conducted systematic reviews based on a priori selected questions. In this exposition, we describe the process used by ASH, including the GRADE approach (Grades of Recommendations, Assessment, Development and Evaluation) for rating certainty of the evidence and the GRADE Evidence to Decision Framework. We also describe several unique challenges faced by the guideline panels and the specific innovations and solutions used to address them, including a curriculum to train patients to engage in guideline development, dealing with the opioid crisis, and working with indirect and noncomparative evidence.
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http://dx.doi.org/10.1182/bloodadvances.2019000931DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6963245PMC
December 2019

American Society of Hematology 2019 guidelines for sickle cell disease: cardiopulmonary and kidney disease.

Blood Adv 2019 12;3(23):3867-3897

Division of Nephrology and Hypertension, School of Medicine, University of Kansas, Kansas City, KS.

Background: Prevention and management of end-organ disease represent major challenges facing providers of children and adults with sickle cell disease (SCD). Uncertainty and variability in the screening, diagnosis, and management of cardiopulmonary and renal complications in SCD lead to varying outcomes for affected individuals.

Objective: These evidence-based guidelines of the American Society of Hematology (ASH) are intended to support patients, clinicians, and other health care professionals in their decisions about screening, diagnosis, and management of cardiopulmonary and renal complications of SCD.

Methods: ASH formed a multidisciplinary guideline panel that included 2 patient representatives and was balanced to minimize potential bias from conflicts of interest. The Mayo Evidence-Based Practice Research Program supported the guideline development process, including performing systematic evidence reviews up to September 2017. The panel prioritized clinical questions and outcomes according to their importance for clinicians and patients. The panel used the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, including GRADE evidence-to-decision frameworks, to assess evidence and make recommendations, which were subject to public comment.

Results: The panel agreed on 10 recommendations for screening, diagnosis, and management of cardiopulmonary and renal complications of SCD. Recommendations related to anticoagulation duration for adults with SCD and venous thromboembolism were also developed.

Conclusions: Most recommendations were conditional due to a paucity of direct, high-quality evidence for outcomes of interest. Future research was identified, including the need for prospective studies to better understand the natural history of cardiopulmonary and renal disease, their relationship to patient-important outcomes, and optimal management.
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http://dx.doi.org/10.1182/bloodadvances.2019000916DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6963257PMC
December 2019

Identifying Clinical and Research Priorities in Sickle Cell Lung Disease. An Official American Thoracic Society Workshop Report.

Ann Am Thorac Soc 2019 09;16(9):e17-e32

Pulmonary complications of sickle cell disease (SCD) are diverse and encompass acute and chronic disease. The understanding of the natural history of pulmonary complications of SCD is limited, no specific therapies exist, and these complications are a primary cause of morbidity and mortality. We gathered a multidisciplinary group of pediatric and adult hematologists, pulmonologists, and emergency medicine physicians with expertise in SCD-related lung disease along with an SCD patient advocate for an American Thoracic Society-sponsored workshop to review the literature and identify key unanswered clinical and research questions. Participants were divided into four subcommittees on the basis of expertise: ) acute chest syndrome, ) lower airways disease and pulmonary function, ) sleep-disordered breathing and hypoxia, and ) pulmonary vascular complications of SCD. Before the workshop, a comprehensive literature review of each subtopic was conducted. Clinically important questions were developed after literature review and were finalized by group discussion and consensus. Current knowledge is based on small, predominantly observational studies, few multicenter longitudinal studies, and even fewer high-quality interventional trials specifically targeting the pulmonary complications of SCD. Each subcommittee identified the three or four most important unanswered questions in their topic area for researchers to direct the next steps of clinical investigation. Important and clinically relevant questions regarding sickle cell lung disease remain unanswered. High-quality, multicenter, longitudinal studies and randomized clinical trials designed and implemented by teams of multidisciplinary clinician-investigators are needed to improve the care of individuals with SCD.
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http://dx.doi.org/10.1513/AnnalsATS.201906-433STDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6812163PMC
September 2019

Nucleated Red Blood Cells in Children With Sickle Cell Disease Hospitalized for Pain.

J Pediatr Hematol Oncol 2019 Nov;41(8):e487-e492

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

Acute chest syndrome (ACS) and transfusion requirements are common and difficult to predict during hospitalizations for acute vaso-occlusive episodes (VOE) among individuals with sickle cell disease (SCD). This study examined the relationship between nucleated red blood cell (NRBC) counts during hospitalization for VOE and development of ACS or transfusion requirement among children with SCD. Retrospective chart review was performed for 264 encounters of patients with SCD hospitalized for uncomplicated VOE who had NRBC count data at admission during a 5-year period. Multivariable logistic regression analysis was conducted to determine the relationship of admission and change in NRBC ([INCREMENT]NRBC) to ACS/transfusion requirement. Overall, 44 of 264 (16.7%) encounters resulted in ACS, transfusion, or both. Admission NRBC was not associated with development of ACS/transfusion requirement. Among 125 of 264 (47.3%) encounters in which a subsequent CBC was obtained, greater increases in NRBCs and greater decrease in hemoglobin were significantly associated with ACS/transfusion requirement (OR, 2.72; 95% CI, 1.16, 6.35; P=0.02 and OR, 2.52; 95% CI, 1.08, 5.89; P=0.03, respectively). Our finding that an increase in NRBC counts was associated with development of ACS/transfusion requirement suggests that [INCREMENT]NRBCs may represent a useful biomarker for predicting complications in children with SCD hospitalized for VOE.
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http://dx.doi.org/10.1097/MPH.0000000000001467DOI Listing
November 2019

Transition to adulthood and adult health care for patients with sickle cell disease or cystic fibrosis: Current practices and research priorities.

J Clin Transl Sci 2018 Oct;2(5):334-342

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

Introduction: A growing population of adults living with severe, chronic childhood-onset health conditions has created a need for specialized health care delivered by providers who have expertise both in adult medicine and in those conditions. Optimal care of these patients requires systematic approaches to healthcare transition (HCT). Guidelines for HCT exist, but gaps in care occur, and there are limited data on outcomes of HCT processes.

Methods: The Single Disease Workgroup of the Lifespan Domain Task Force of the National Center for Advancing Translational Sciences Clinical and Translational Science Award programs convened a group to review the current state of HCT and to identify gaps in research and practice. Using cystic fibrosis and sickle cell disease as models, key themes were developed. A literature search identified general and disease-specific articles. We summarized key findings.

Results: We identified literature characterizing patient, parent and healthcare provider perspectives, recommendations for transition care, and barriers to effective transition.

Conclusions: With increased survival of patients with severe childhood onset diseases, ongoing study of effective transition practices is essential as survival increases for severe childhood onset diseases. We propose pragmatic methods to enhance transition research to improve health and key outcomes.
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http://dx.doi.org/10.1017/cts.2018.338DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6390387PMC
October 2018

Healthcare utilization and hydroxyurea adherence in youth with sickle cell disease.

Pediatr Hematol Oncol 2018 Aug - Sep;35(5-6):297-308. Epub 2019 Jan 12.

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

Background: Sickle cell disease (SCD) complications lead to poor health-related quality of life (HRQOL) and increased healthcare utilization in this population, which could be mitigated with hydroxyurea therapy; however, adherence is suboptimal. We assessed the relationship of healthcare utilization to hydroxyurea adherence and HRQOL amongst youth with SCD.

Methods: Thirty-four patients with SCD (median age 14 years, IQR 12-18) on hydroxyurea participated in this cross-sectional study and completed Morisky Adherence Scale 8-items and Patient Reported Outcomes Measurement Information System (PROMIS®) HRQOL measures. A medical chart review was conducted to assess healthcare utilization.

Results: Participants with more frequent hospitalizations and emergency room (ER) visits and longer length of stay (LOS) had significantly lower fetal hemoglobin (r=-0.44; r=-0.45; r=-0.46, p < 0.05) and mean corpuscular volume (r=-0.47; r=-0.42; r=-0.48, p < 0.05), respectively. More frequent hospitalizations and ER visits and longer LOS correlated significantly with worse fatigue (r=0.51; r=0.41; r=0.53, p < 0.05), pain (r=0.41; r=0.38; r=0.47, p < 0.05), physical function mobility (r=-0.67; r=-0.59; r=-0.67, p < 0.05), depression (r=0.38; r=0.31; r=0.42, p < 0.05), and social isolation (r=0.76; r=0.76; r=-0.84, p < 0.05), respectively.

Conclusions: We conclude that increased healthcare utilization in youth with SCD is associated with low adherence to hydroxyurea and worse HRQOL domain scores. It is important emphasize the clinical benefits of high adherence to hydroxyurea, particularly among youth with SCD. Future longitudinal studies are warranted to assess the directionality of these relationships, and may reveal modifiable behavioral factors associated with early changes in hydroxyurea adherence levels.
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http://dx.doi.org/10.1080/08880018.2018.1505988DOI Listing
March 2019

Balancing exercise risk and benefits: lessons learned from sickle cell trait and sickle cell anemia.

Authors:
Robert I Liem

Hematology Am Soc Hematol Educ Program 2018 11;2018(1):418-425

Hematology, Oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; and Department of Pediatrics, Feinberg School of Medicine, Northwestern University, Chicago, IL.

Development of exercise guidelines for individuals with sickle cell trait (SCT) and sickle cell anemia (SCA) is hampered by the need to weigh the benefits against risks of exercise in these populations. In SCT, concern for exercise collapse associated with sickle cell trait has resulted in controversial screening of student athletes for SCT. In SCA, there exists unsubstantiated concerns that high-intensity exercise may result in pain and other complications. In both, finding the "right dose" of exercise remains a challenge for patients and their providers. Despite assumptions that factors predisposing to adverse events from high-intensity exercise overlap in SCT and SCA, the issues that frame our understanding of exercise-related harms in both are distinct. This review will compare issues that affect the risk-benefit balance of exercise in SCT and SCA through these key questions: (1) What is the evidence that high-intensity exercise is associated with harm? (2) What are the pathophysiologic mechanisms that could predispose to harm? (3) What are the preventive strategies that may reduce risk? and (4) Why do we need to consider the benefits of exercise in this debate? Addressing these knowledge gaps is essential for developing an evidence-based exercise prescription for these patient populations.
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http://dx.doi.org/10.1182/asheducation-2018.1.418DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6245992PMC
November 2018

Exercise capacity and clinical outcomes in adults followed in the Cooperative Study of Sickle Cell Disease (CSSCD).

Eur J Haematol 2018 Oct 31;101(4):532-541. Epub 2018 Aug 31.

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

Objectives: To determine the factors associated with exercise capacity in adults with sickle cell disease (SCD) and its relationship to hospitalizations and mortality.

Methods: A total of 223 participants in the Cooperative Study of Sickle Cell Disease (CSSCD) (64% female, 70% hemoglobin SS/Sβ thalassemia, mean age 43.3 ± 7.5 years) underwent maximal exercise testing using a treadmill protocol with a mean duration of 11.6 ± 5.2 minutes.

Results: Female sex (β = -3.34, 95% CI [-1.80, -4.88], P < 0.001), older age (β = -0.14, 95% CI [-0.24, -0.04], P = 0.005), higher body mass index (β = -0.23, 95% CI [-0.37, -0.10]; P = 0.001), and lower hemoglobin (β = 0.56, 95% CI [0.08, 1.04], P = 0.02) were independently associated with lower fitness, while there was a trend with abnormal pulmonary function testing (β = -1.42, 95% CI [-2.92, 0.07]; P = 0.06). Lower percent-predicted forced expiratory volume in 1 second (FEV ) was independently associated with lower fitness (β = 0.08, 95% CI [0.03, 0.13], P = 0.001). Genotype and hospitalization rates for pain and acute chest syndrome (ACS) prior to testing were not associated with exercise capacity. Baseline exercise capacity predicted neither future pain or ACS nor survival in our cohort. Adults with SCD tolerated maximal exercise testing.

Conclusions: Prospective studies are needed to further evaluate the impact of regular exercise and improved fitness on clinical outcomes and mortality in SCD.
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http://dx.doi.org/10.1111/ejh.13140DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6546160PMC
October 2018

Risk Factors for Neonatal Venous and Arterial Thromboembolism in the Neonatal Intensive Care Unit-A Case Control Study.

J Pediatr 2018 04 2;195:28-32. Epub 2018 Feb 2.

Division of Hematology, Oncology and Stem Cell Transplant, Department of Pediatrics, Ann & Robert H. Lurie Children's Hospital, Northwestern University Feinberg School of Medicine, Chicago, IL.

Objective: To identify risk factors associated with venous and arterial thrombosis in sick neonates admitted to the neonatal intensive care unit.

Study Design: A case-control study was conducted at 2 centers between January 2010 and March 2014 using the Children's Hospital Neonatal Database dataset. Cases were neonates diagnosed with either arterial or venous thrombosis during their neonatal intensive care unit stay; controls were matched in a 1:4 ratio by gestational age and presence or absence of central access devices. Bivariable and conditional logistic regression analyses for venous and arterial thrombosis were performed separately.

Results: The overall incidence of neonatal thrombosis was 15.0 per 1000 admissions. A higher proportion of neonates with thrombosis had presence of central vascular access devices (75% vs 49%; P < .01) were of extremely preterm gestational age (22-27 weeks; 26% vs 15.0%; P <.05) and stayed ≥31 days in the neonatal intensive care unit (53% vs 32.9%; P <.01), when compared with neonates without thrombosis. A final group of 64 eligible patients with thrombosis and 4623 controls were analyzed. In a conditional multivariable logistic regression model, venous thrombosis was significantly associated with male sex (AOR, 2.12; 95% CI, 1.03-4.35; P = .04) and blood stream infection (AOR, 3.47; 95% CI, 1.30-9.24; P = .01).

Conclusions: The incidence of thrombosis was higher in our neonatal population than in previous reports. After matching for central vascular access device and gestational age, male sex and blood stream infection represent independent risk factors of neonatal venous thrombosis. A larger cohort gleaned from multicenter data should be used to confirm the study results and to develop thrombosis prevention strategies.
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http://dx.doi.org/10.1016/j.jpeds.2017.12.015DOI Listing
April 2018

Beliefs about hydroxyurea in youth with sickle cell disease.

Hematol Oncol Stem Cell Ther 2018 Sep 2;11(3):142-148. Epub 2018 Feb 2.

Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL, USA; Division of Hematology, Oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital, Chicago, IL, USA.

Background: Hydroxyurea reduces complications and improves health-related quality of life (HRQOL) in sickle cell disease (SCD) patients, however adherence remains suboptimal. Understanding patients' views of hydroxyurea is critical to optimize adherence, particularly in adolescents and young adults (AYA). Study objectives were to assess beliefs about hydroxyurea using the Beliefs about Medicines Questionnaire (BMQ), and to examine the relationship of patients' beliefs to their hydroxyurea adherence and HRQOL.

Methods: Thirty-four AYA with SCD participated in a cross-sectional study January-December 2015. Study assessments included BMQ to examine beliefs about hydroxyurea; Visual Analogue Scale (VAS) to assess hydroxyurea adherence; and Patient Reported Outcomes Measurement Information System (PROMIS®) to evaluate HRQOL.

Results: Participants (41% female, 91% Black) had median age of 13.5 (IQR 12-18) years. Participants' concerns about overuse of medications correlated with concerns about hydroxyurea (r = 0.36, p = 0.04) and overall harm of medications (r = 0.5, p = 0.003). Participants' age positively correlated with the necessity of hydroxyurea (r = 0.45, p = 0.007). Participants' concerns about hydroxyurea and overuse of medications positively correlated with anxiety (r = 0.41, p = 0.02; r = 0.44, p = 0.01) and depression (r = 0.37, p = 0.04; r = 0.54, p = 0.001), but inversely correlated with peer relationships (r = -0.45, p = 0.03; r = -0.44, p = 0.03), respectively, suggesting better HRQOL with concerns. Fifty percent of participants reported low hydroxyurea adherence (VAS < 80%), which was more seen in patients with higher concerns about hydroxyurea (p = 0.02).

Conclusions: Beliefs about hydroxyurea correlated with HRQOL scores and adherence levels. Addressing patients' concern about hydroxyurea and medications overall as well as routine assessment of adherence and beliefs could help to overcome adherence barriers.
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http://dx.doi.org/10.1016/j.hemonc.2018.01.001DOI Listing
September 2018

Adherence to hydroxyurea, health-related quality of life domains, and patients' perceptions of sickle cell disease and hydroxyurea: a cross-sectional study in adolescents and young adults.

Health Qual Life Outcomes 2017 Jul 5;15(1):136. Epub 2017 Jul 5.

Department of Pediatrics, Division of Hematology, Oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital, Northwestern University Feinberg School of Medicine, 225 E. Chicago Ave., Box #30, Chicago, IL, 60611, USA.

Background: Sickle cell disease (SCD) patients have impaired domains of health-related quality of life (HRQOL). Hydroxyurea is safe and efficacious in SCD; however, adherence is suboptimal, and patients' perceptions are poorly understood amongst adolescents and young adults (AYA). Study objectives were to: (1) examine patients' perceptions of SCD and hydroxyurea; and (2) explore the relationship of their perceptions to clinical characteristics, HRQOL domains and hydroxyurea adherence.

Methods: Thirty-four SCD patients on hydroxyurea (≥6 months) participated in a single-institution study. Study measures included Brief-Illness Perceptions Questionnaire, ©Modified Morisky Adherence Scale 8-items, and Patient Reported Outcomes Measurement Information System (PROMIS®). We assessed the relationship of patients' perceptions to hydroxyurea adherence using Wilcoxon rank-sum test, the number of hospitalizations using Kruskal-Wallis test, and the number of ED visits, adherence level, HRQOL domain scores using Spearman's rho correlations. We conducted a sub-analysis in HbSS patients to evaluate the relationship of patients' perceptions to laboratory markers of hydroxyurea adherence.

Results: Participants were 59% male and 91% Black, and had a median age of 13.5 (range 12-18) years. Participants with ≥4 hospitalizations over 1-year prior (using electronic medical chart review) reported more negative perceptions of SCD-related symptoms and emotional response, and perceived hydroxyurea as less beneficial; all p-values ≤0.01. Most participants (74%) reported low hydroxyurea adherence. Participants with higher hydroxyurea adherence perceived more hydroxyurea benefits (r  = 0.44, p < 0.01) and had better emotional response to SCD (r  = -0.44, p = 0.01). In a sub-analysis of HbSS patients, perceived benefits of hydroxyurea positively correlated with HbF (r  = 0.37, p = 0.05) and MCV values (r  = 0.35, p = 0.05). Participants with more negative perceptions of SCD-related consequences, concerns, and emotional response, and fewer perceived hydroxyurea benefits reported worse fatigue (r  = 0.68; r  = 0.44; r  = 0.74; r  = -0.60), pain (r  = 0.56; r  = 0.54; r  = 0.63; r  = -0.39), anxiety (r  = 0.55; r  = 0.58; r  = 0.56; r  = -0.47), and depression (r  = 0.64; r  = 0.49; r  = 0.70; r  = -0.62), respectively, all p-values <0.05.

Conclusions: Dynamics influencing hydroxyurea adherence are multifactorial, and understanding patients' perceptions is critical to overcoming adherence barriers. Patients' favorable perceptions correlated with greater adherence and better HRQOL domain scores. Prospective evaluation of patients' perceptions of SCD and hydroxyurea in relation adherence, HRQOL domains and clinical outcomes is warranted.
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http://dx.doi.org/10.1186/s12955-017-0713-xDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5498866PMC
July 2017

Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module.

Health Qual Life Outcomes 2017 Jun 12;15(1):124. Epub 2017 Jun 12.

Medical College of Wisconsin, Pediatric Emergency Medicine, and the Children's Hospital of Wisconsin, Milwaukee, WI, USA.

Background: Detecting change in health status over time and ascertaining meaningful changes are critical elements when using health-related quality of life (HRQL) instruments to measure patient-centered outcomes. The PedsQL™ Sickle Cell Disease module, a disease specific HRQL instrument, has previously been shown to be valid and reliable. Our objectives were to determine the longitudinal validity of the PedsQL™ Sickle Cell Disease module and the change in HRQL that is meaningful to patients.

Methods: An ancillary study was conducted utilizing a multi-center prospective trial design. Children ages 4-21 years with sickle cell disease admitted to the hospital for an acute painful vaso-oclusive crisis were eligible. Children completed HRQL assessments at three time points (in the Emergency Department, one week post-discharge, and at return to baseline (One to three months post-discharge). The primary outcome was change in HRQL score. Both distribution (effect size, standard error of measurement (SEM)) and anchor (global change assessment) based methods were used to determine the longitudinal validity and meaningful change in HRQL. Changes in HRQL meaningful to patients were identified by anchoring the change scores to the patient's perception of global improvement in pain.

Results: Moderate effect sizes (0.20-0.80) were determined for all domains except the Communication I and Cognitive Fatigue domains. The value of 1 SEM varied from 3.8-14.6 across all domains. Over 50% of patients improved by at least 1 SEM in Total HRQL score. A HRQL change score of 7-10 in the pain domains represented minimal perceived improvement in HRQL and a HRQL change score of 18 or greater represented moderate to large improvement.

Conclusions: The PedsQL™ Sickle Cell Disease Module is responsive to changes in HRQL in patients experiencing acute painful vaso-occlusive crises. The study data establish longitudinal validity and meaningful change parameters for the PedsQL™ Sickle Cell Disease Module.

Trial Registration: ClinicalTrials.gov (study identifier: NCT01197417 ). Date of registration: 08/30/2010.
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http://dx.doi.org/10.1186/s12955-017-0700-2DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5468970PMC
June 2017

Feasibility and safety of home exercise training in children with sickle cell anemia.

Pediatr Blood Cancer 2017 Dec 9;64(12). Epub 2017 Jun 9.

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

Exercise guidelines do not exist for individuals with sickle cell anemia (SCA) despite the impact of disease-related complications on physical functioning. Thirteen subjects (mean 15.1 ± 2.8 years old) with SCA were prescribed three exercise sessions/week for 12 weeks on a stationary bicycle placed at home. In total, 77% of subjects completed 89% of prescribed sessions without exercise-related adverse events, thus meeting feasibility and safety criteria. Adherence to prescribed duration and target heart rate during training decreased during the second half of the study. Future trials are warranted to further evaluate training benefits associated with regular exercise in children with SCA.
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http://dx.doi.org/10.1002/pbc.26671DOI Listing
December 2017

Self-Reported Physical Activity and Exercise Patterns in Children With Sickle Cell Disease.

Pediatr Exerc Sci 2017 08 22;29(3):388-395. Epub 2017 May 22.

1 Ann & Robert H. Lurie Children's Hospital of Chicago.

Purpose: Sickle cell disease (SCD) significantly affects physical functioning. We examined physical activity (PA) patterns in children with SCD versus a national sample and factors associated with PA and participation in physical education and organized sports.

Method: One hundred children with SCD completed a 58-item survey with questions from the 2009-2010 National Health and Nutrition Examination Survey (NHANES) Physical Activity Questionnaire and others on physical education and sports, disease impact, and physical functioning.

Results: Compared with NHANES participants, more children with SCD (67 vs 42%, p < .01) reported doing at least 10 min of moderate-to-vigorous intensity PA (MVPA)/week. Children with SCD also reported spending more days in MVPA (2.3 vs. 1.4 days/week, p < .01). However, fewer reported spending ³ 60 min/day in either vigorous PA (VPA) (24 vs. 43%, p = .01) or MVPA (17 vs 23%, p < .01). In addition, 90% and 48% of children with SCD participated in physical education and sports, respectively. Greater disease impact on PA and physical functioning were associated with lower participation.

Conclusion: Children with SCD are active at moderate to vigorous intensity for shorter durations. Negative personal beliefs about disease impact and poor physical functioning represent barriers to PA in SCD.
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http://dx.doi.org/10.1123/pes.2016-0276DOI Listing
August 2017

Red blood cell transfusions during sickle cell anemia vaso-occlusive crises: a report from the magnesium in crisis (MAGiC) study.

Transfusion 2017 08 12;57(8):1891-1897. Epub 2017 May 12.

Department of Pediatrics, Medical College of Wisconsin, Milwaukee, Wisconsin.

Background: Little is known about red blood cell (RBC) transfusion practices for children hospitalized for a sickle cell vaso-occlusive pain crisis (VOC). We hypothesized that transfusion would be associated with the development of acute chest syndrome (ACS), lower hemoglobin (Hb) concentration, and lack of hydroxyurea therapy.

Study Design And Methods: This is a secondary analysis of all children admitted for a sickle cell pain crisis enrolled in the Magnesium in Crisis (MAGiC) randomized trial; all had HbSS or S-β thalassemia. ACS development and transfusion administration were prospectively collected during the parent trial. All Hb values during the hospitalization were recorded, as was parent report of child receiving hydroxyurea. Relative risks (RRs) of transfusion were compared between groups.

Results: Of 204 enrolled children, 40 (19.6%) received a transfusion. Of the 30 children who developed ACS, 22 (73.3%) received transfusions compared to 18 of 174 (10.3%) without ACS: the RR of transfusion in children with ACS was 7.1 (95% confidence interval [CI], 4.4-11.5). Among those without ACS, the lowest Hb was most strongly associated with transfusions: RR was 3.1 (95% CI 2.0 - 4.7) for each 1 g/dL decrease in lowest Hb. In a binary recursive partitioning model for those without ACS, a lowest recorded Hb level of less than 6.3 g/dL was significantly associated with transfusion during admission (p < 0.01). Hydroxyurea use was not associated with transfusions in any analysis.

Conclusion: ACS increased the RR of transfusion in children hospitalized for VOC sevenfold. In children without ACS, transfusion was associated with lowest Hb concentration, particularly Hb concentration of less than 6.3 g/dL.
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http://dx.doi.org/10.1111/trf.14155DOI Listing
August 2017

Barriers to hydroxyurea adherence and health-related quality of life in adolescents and young adults with sickle cell disease.

Eur J Haematol 2017 Jun 17;98(6):608-614. Epub 2017 Apr 17.

Department of Pediatrics, Ann & Robert H. Lurie Children's Hospital, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.

Objectives: To identify barriers to hydroxyurea adherence (negative beliefs, access, and/or recall barriers), and their relationship to adherence rates and health-related quality of life (HRQOL) among adolescents and young adults (AYA) with sickle cell disease (SCD).

Methods: A cross-sectional survey was administered to 34 AYAs (12-22 years old) in SCD clinics from January to December 2015. Study measures included Brief Medication Questionnaire, Modified Morisky Adherence Scale 8-items, visual analog scale, and Patient Reported Outcomes Measurement Information System.

Results: Participants (59% male; 91% Black) had a median age of 13.5 years (IQR 12-18). Participants reported negative beliefs (32%), recall barriers (44%), and access barriers (32%). Participants with recall barriers reported worse pain (P=.02), fatigue (P=.05), and depression (P=.05). The number of adherence barriers inversely correlated with adherence level using ©MMAS-8 (r =-.38, P=.02) and VAS (r =-.25, P=.14) as well as MCV (r =-.45, P=.01) and HbF% (r =-.36, P=.05), suggesting higher hydroxyurea adherence in patients with fewer barriers.

Conclusions: Patients with fewer barriers to hydroxyurea adherence were more likely to have higher adherence rates and better HRQOL scores. Routine assessment of hydroxyurea adherence and its related barriers could provide actionable information to improve adherence rates, HRQOL, and other clinical outcomes.
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http://dx.doi.org/10.1111/ejh.12878DOI Listing
June 2017

Association of Sickle Cell Trait With Hemoglobin A1c in African Americans.

JAMA 2017 02;317(5):507-515

Department of Epidemiology, Brown University School of Public Health, Providence, Rhode Island13Center for Innovation in Long-Term Services and Support, Providence Veterans Affairs Medical Center, Providence, Rhode Island14Division of Cardiology, Providence Veterans Affairs Medical Center, Providence, Rhode Island15Department of Medicine, Alpert Medical School, Brown University, Providence, Rhode Island.

Importance: Hemoglobin A1c (HbA1c) reflects past glucose concentrations, but this relationship may differ between those with sickle cell trait (SCT) and those without it.

Objective: To evaluate the association between SCT and HbA1c for given levels of fasting or 2-hour glucose levels among African Americans.

Design, Setting, And Participants: Retrospective cohort study using data collected from 7938 participants in 2 community-based cohorts, the Coronary Artery Risk Development in Young Adults (CARDIA) study and the Jackson Heart Study (JHS). From the CARDIA study, 2637 patients contributed a maximum of 2 visits (2005-2011); from the JHS, 5301 participants contributed a maximum of 3 visits (2000-2013). All visits were scheduled at approximately 5-year intervals. Participants without SCT data, those without any concurrent HbA1c and glucose measurements, and those with hemoglobin variants HbSS, HbCC, or HbAC were excluded. Analysis of the primary outcome was conducted using generalized estimating equations (GEE) to examine the association of SCT with HbA1c levels, controlling for fasting or 2-hour glucose measures.

Exposures: Presence of SCT.

Main Outcomes And Measures: Hemoglobin A1c stratified by the presence or absence of SCT was the primary outcome measure.

Results: The analytic sample included 4620 participants (mean age, 52.3 [SD, 11.8] years; 2835 women [61.3%]; 367 [7.9%] with SCT) with 9062 concurrent measures of fasting glucose and HbA1c levels. In unadjusted GEE analyses, for a given fasting glucose, HbA1c values were statistically significantly lower in those with (5.72%) vs those without (6.01%) SCT (mean HbA1c difference, -0.29%; 95% CI, -0.35% to -0.23%). Findings were similar in models adjusted for key risk factors and in analyses using 2001 concurrent measures of 2-hour glucose and HbA1c concentration for those with SCT (mean, 5.35%) vs those without SCT (mean, 5.65%) for a mean HbA1c difference of -0.30% (95% CI, -0.39% to -0.21%). The HbA1c difference by SCT was greater at higher fasting (P = .02 for interaction) and 2-hour (P = .03) glucose concentrations. The prevalence of prediabetes and diabetes was statistically significantly lower among participants with SCT when defined using HbA1c values (29.2% vs 48.6% for prediabetes and 3.8% vs 7.3% for diabetes in 572 observations from participants with SCT and 6877 observations from participants without SCT; P<.001 for both comparisons).

Conclusions And Relevance: Among African Americans from 2 large, well-established cohorts, participants with SCT had lower levels of HbA1c at any given concentration of fasting or 2-hour glucose compared with participants without SCT. These findings suggest that HbA1c may systematically underestimate past glycemia in black patients with SCT and may require further evaluation.
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http://dx.doi.org/10.1001/jama.2016.21035DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5713881PMC
February 2017

Pediatric Exercise Testing: Value and Implications of Peak Oxygen Uptake.

Children (Basel) 2017 Jan 24;4(1). Epub 2017 Jan 24.

EMGO + Institute for Health and Care Research, VU University Medical Center, 1081 Amsterdam, The Netherlands.

Peak oxygen uptake (peak V ˙ O 2 ) measured by clinical exercise testing is the benchmark for aerobic fitness. Aerobic fitness, estimated from maximal treadmill exercise, is a predictor of mortality in adults. Peak V ˙ O 2 was shown to predict longevity in patients aged 7-35 years with cystic fibrosis over 25 years ago. A surge of exercise studies in young adults with congenital heart disease over the past decade has revealed significant prognostic information. Three years ago, the first clinical trial in children with pulmonary arterial hypertension used peak V ˙ O 2 as an endpoint that likewise delivered clinically relevant data. Cardiopulmonary exercise testing provides clinicians with biomarkers and clinical outcomes, and researchers with novel insights into fundamental biological mechanisms reflecting an integrated physiological response hidden at rest. Momentum from these pioneering observations in multiple disease states should impel clinicians to employ similar methods in other patient populations; e.g., sickle cell disease. Advances in pediatric exercise science will elucidate new pathways that may identify novel biomarkers. Our initial aim of this essay is to highlight the clinical relevance of exercise testing to determine peak V ˙ O 2 , and thereby convince clinicians of its merit, stimulating future clinical investigators to broaden the application of exercise testing in pediatrics.
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http://dx.doi.org/10.3390/children4010006DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5296667PMC
January 2017

Health-related quality of life and adherence to hydroxyurea in adolescents and young adults with sickle cell disease.

Pediatr Blood Cancer 2017 06 28;64(6). Epub 2016 Nov 28.

Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, Illinois.

Background: Complications related to sickle cell disease (SCD) result in significant declines in health-related quality of life (HRQOL). While hydroxyurea reduces SCD complications, adherence remains suboptimal. The study's objectives were to assess the feasibility of Internet-based electronic assessment of HRQOL in SCD clinic and to examine the relationship between HRQOL and hydroxyurea adherence in adolescents and young adults (AYAs) with SCD.

Procedure: A cross-sectional survey was administered on tablets to 34 AYAs (12-22 years old) in a SCD clinic from January through December 2015. Study measures included Patient Reported Outcomes Measurement Information System (PROMIS ) computerized adaptive testing and ©Modified Morisky Adherence Scale 8-items (©MMAS-8).

Results: Participants (59% male, 91% Black) had median age of 13.5 (range 12-18) years. Ninety-one percent completed PROMIS® measures electronically in the clinic, meeting our feasibility criterion of ≥85% completion rate. ©MMAS-8 scores positively correlated with fetal hemoglobin (HbF) (r = 0.34, P = 0.04) and mean corpuscular volume (MCV) (r = 0.42, P = 0.01) and inversely correlated with fatigue (r = -0.45, P = 0.01), depression (r = -0.3, P = 0.08), and social isolation (r = -0.78, P = 0.02). Low ©MMAS-8 scores, indicating poor adherence, were associated with worse fatigue (P = 0.001) and trended toward significance for pain (P = 0.07) and depression (P = 0.06). Homozygous hemoglobin S disease patients with low HbF (<16%) had worse social isolation (P = 0.04) and those with low MCV (<102 fl) reported worse fatigue (P = 0.001), pain (P = 0.01), mobility (P = 0.01), and social isolation (P = 0.04).

Conclusions: HRQOL assessment in the SCD clinic is feasible. SCD patients with low hydroxyurea adherence and/or low HbF or MCV levels had worse HRQOL scores, particularly fatigue. Future prospective studies examining the relationship between HRQOL and hydroxyurea adherence are warranted.
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http://dx.doi.org/10.1002/pbc.26369DOI Listing
June 2017

Association among sickle cell trait, fitness, and cardiovascular risk factors in CARDIA.

Blood 2017 02 16;129(6):723-728. Epub 2016 Nov 16.

Department of Preventive Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL; and.

The contribution of sickle cell trait (SCT) to racial disparities in cardiopulmonary fitness is not known, despite concerns that SCT is associated with exertion-related sudden death. We evaluated the association of SCT status with cross-sectional and longitudinal changes in fitness and risk for hypertension, diabetes, and metabolic syndrome over the course of 25 years among 1995 African Americans (56% women, 18-30 years old) in the Coronary Artery Risk Development in Young Adults (CARDIA) study. Overall, the prevalence of SCT was 6.8% (136/1995) in CARDIA, and over the course of 25 years, 46% (738/1590), 18% (288/1631), and 40% (645/1,611) of all participants developed hypertension, diabetes, and metabolic syndrome, respectively. Compared with participants without SCT, participants with SCT had similar baseline measures of fitness in cross-section, including exercise duration (535 vs 540 seconds; = .62), estimated metabolic equivalent of tasks (METs; 11.6 vs 11.7; = .80), maximum heart rate (174 vs 175 beats/min; = .41), and heart rate at 2 minutes recovery (44 vs 43 beats/min; = .28). In our secondary analysis, there was neither an association of SCT status with longitudinal changes in fitness nor an association with development of hypertension, diabetes, or metabolic syndrome after adjustment for sex, baseline age, body mass index, fitness, and physical activity. SCT is not associated with reduced fitness in this longitudinal study of young African American adults, suggesting the increased risk for exertion-related sudden death in SCT carriers is unlikely related to fitness. SCT status also is not an independent risk factor for developing hypertension, diabetes, or metabolic syndrome.
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http://dx.doi.org/10.1182/blood-2016-07-727719DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5301825PMC
February 2017

Opioid prescription practices at discharge and 30-day returns in children with sickle cell disease and pain.

Pediatr Blood Cancer 2017 05 1;64(5). Epub 2016 Nov 1.

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

Background: Acute pain episodes in children with sickle cell disease (SCD) represent a leading cause of readmissions. We examined prescription practices at the time of discharge in children with SCD presenting with acute pain to determine their impact on 30-day emergency department (ED) revisits and readmissions.

Methods: In this single-institution, 5-year retrospective study, we reviewed 290 encounters of patients with SCD aged 7-21 years hospitalized or discharged from the ED with acute pain. We reviewed demographic, treatment and discharge data, and 30-day returns, defined as ED revisits and readmissions within 30 days of discharge. Bivariate and multivariable analyses were performed to evaluate the association between discharge prescription practices and 30-day returns.

Results: Compared to hospitalizations, treat-and-release ED visits for acute pain were associated with a higher incidence of 30-day returns (OR = 2.7 [95% CI: 1.5-4.8], P < 0.01). We found no association between prescribed opioid frequency (scheduled vs. as-needed) and 30-day returns (OR = 1.12 [95% CI: 0.62-2.02], P = 0.70). By multivariable logistic regression, the prescription of nonsteroidal anti-inflammatory drugs (NSAIDs) only, without opioids, after treat-and-release ED visits was independently associated with a higher frequency of 30-day ED revisits (OR = 6.9 [95% CI: 1.3-37.3], P = 0.03) but not readmissions.

Conclusion: Variability exists in opioid prescription practices after discharge in children with SCD and pain episodes. Prescription of NSAIDs only, without opioids, was an independent predictor of higher 30-day ED revisits. Formalized studies to better understand factors that influence returns, including outpatient opioid management, are warranted in this population.
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http://dx.doi.org/10.1002/pbc.26319DOI Listing
May 2017

Assessing cardiac and liver iron overload in chronically transfused patients with sickle cell disease.

Br J Haematol 2016 Nov 10;175(4):705-713. Epub 2016 Aug 10.

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

Transfusional iron overload represents a substantial challenge in the management of patients with sickle cell disease (SCD) who receive chronic or episodic red blood cell transfusions. Iron-induced cardiomyopathy is a leading cause of death in other chronically transfused populations but rarely seen in SCD. Study objectives were to: (i) examine the extent of myocardial and hepatic siderosis using magnetic resonance imaging (MRI) in chronically transfused SCD patients, and (ii) evaluate the relationship between long-term (over the 5 years prior to enrolment) mean serum ferritin (MSF), spot-ferritin values and liver iron content (LIC) measured using MRI and liver biopsy. Thirty-two SCD patients (median age 15 years) with transfusional iron overload were recruited from two U.S. institutions. Long-term MSF and spot-ferritin values significantly correlated with LIC by MRI-R2* (r = 0·77, P < 0·001; r = 0·82, P < 0·001, respectively). LIC by MRI-R2* had strong positive correlation with LIC by liver biopsy (r = 0·98, P < 0·001) but modest inverse correlation with cardiac MRI-T2* (r = -0·41, P = 0·02). Moderate to severe transfusional iron overload in SCD was not associated with aberrations in other measures of cardiac function based on echocardiogram or serum biomarkers. Our results suggest that SCD patients receiving chronic transfusions may not demonstrate significant cardiac iron loading irrespective of ferritin trends, LIC and erythropoiesis suppression.
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http://dx.doi.org/10.1111/bjh.14277DOI Listing
November 2016
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