title: Hydroxyurea effectiveness in children and adolescents with sickle cell anemia: A large retrospective, population-based cohort.
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, Sβ0thalassemia) 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. © 2016 Wiley Periodicals, Inc.
authors.affiliation: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia.|Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Maa-Ohui
authors.initials: MO
authors.lastname: Quarmyne
authors.affiliation: Connance, Inc, Waltham, Massachusetts.
authors.forename: Wei
authors.initials: W
authors.lastname: Dong
authors.affiliation: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia.
authors.forename: Rodney
authors.initials: R
authors.lastname: Theodore
authors.affiliation: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia.
authors.forename: Sonia
authors.initials: S
authors.lastname: Anand
authors.affiliation: Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Vaughn
authors.initials: V
authors.lastname: Barry
authors.affiliation: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia.|Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Olufolake
authors.initials: O
authors.lastname: Adisa
authors.affiliation: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia.|Department of Pediatrics, Morehouse School of Medicine, Atlanta, Georgia.
authors.forename: Iris D
authors.initials: ID
authors.lastname: Buchanan
authors.affiliation: Outcomes Center, Children's Healthcare of Atlanta, Atlanta, Georgia.
authors.forename: James
authors.initials: J
authors.lastname: Bost
authors.affiliation: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia.|Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Robert C
authors.initials: RC
authors.lastname: Brown
authors.affiliation: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia.|Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Clinton H
authors.initials: CH
authors.lastname: Joiner
authors.affiliation: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia.|Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Peter A
authors.initials: PA
authors.lastname: Lane
mesh_terms: D000293:Adolescent; D000367:Age Factors; D000755:Anemia, Sickle Cell / Q000097:blood / Q000188:drug therapy*; D000986:Antisickling Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D002648:Child; D015331:Cohort Studies; D005260:Female; D006760:Hospitalization / Q000706:statistics & numerical data / Q000639:trends*; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage / Q000627:therapeutic use*; D008297:Male; D008499:Medical Records; D012189:Retrospective Studies; D016896:Treatment Outcome
chemical_list: D000986:Antisickling Agents; D006918:Hydroxyurea