Hydroxyurea effectiveness in children and adolescents with sickle cell anemia: A large retrospective, population-based cohort

• Type of Material: Article
• Authors: Maa-Ohui Quarmyne, Emory University; Wei Dong, Connance, Inc; Rodney Theodore, Children's Healthcare of Atlanta; Sonia Anand, Children's Healthcare of Atlanta; Vaughn Barry, Emory University; Fola Adisa, Emory University; Iris D. Buchanan, Children's Healthcare of Atlanta; James Bost, Children's Healthcare of Atlanta; Robert C. Brown, Children's Healthcare of Atlanta; Clinton Joiner, Emory University; Peter Lane, Emory University
• Language: English
• Date: 2017-01-01
• Publisher: Wiley
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2016 Wiley Periodicals, Inc.
• Final Published Version (URL): http://dx.doi.org/10.1002/ajh.24587
• Title of Journal or Parent Work: American Journal of Hematology
• ISSN: 0361-8609
• Volume: 92
• Issue: 1
• Start Page: 77
• End Page: 81
• Grant/Funding Information: Supported by the National Center for Advancing Translational Sciences of the National Institutes of Health under Award Number UL1TR000454.; This work was also supported by a grant from the Abraham J. & Phyllis Katz Foundation.; The content is solely the responsibility of the authors and does not necessarily represent official view of the National Institutes of Health.
• Keywords: IMPROVED SURVIVAL; RISK; Science & Technology; TRANSPLANTATION; CONTROLLED-TRIAL; DISEASE; Life Sciences & Biomedicine; TRANSFUSIONS; MORTALITY; STROKE; Hematology; MORBIDITY; DEATH
• Research Categories: Health Sciences, Medicine and Surgery; Health Sciences, Epidemiology

Abstract

The clinical efficacy of hydroxyurea in patients with sickle cell anemia (SCA) has been well established. However, data about its clinical effectiveness in practice is limited. We evaluated the clinical effectiveness of hydroxyurea in a large pediatric population using a retrospective cohort, pre-post treatment study design to control for disease severity selection bias. The cohort included children with SCA (SS, Sβ(0) thalassemia) who received care at Children's Healthcare of Atlanta (CHOA) and who initiated hydroxyurea in 2009-2011. Children on chronic transfusions, or children with inadequate follow up data and/or children who had taken hydroxyurea in the 3 years prior were excluded. For each patient healthcare utilization, laboratory values, and clinical outcomes for the 2-year period prior to hydroxyurea initiation were compared to those 2 years after initiation. Of 211 children with SCA who initiated hydroxyurea in 2009-2011, 134 met eligibility criteria. After initiation of hydroxyurea, rates of hospitalizations, pain encounters, and emergency department visits were reduced by 47% (<0.0001), 36% (P = 0.0001) and 43% (P < 0.0001), respectively. Average hemoglobin levels increased by 0.7 g/dl (P < 0.0001). Hydroxyurea effectiveness was similar across gender, insurance types and age, although there was a slightly greater reduction in hospitalizations in younger children. Am. J. Hematol. 92:77-81, 2017.