Ten-year Longitudinal Analysis of Hydroxyurea Implementation in a Pediatric Sickle Cell Program
Document Type
Journal Article
Publication Date
7-10-2022
Journal
European journal of haematology
DOI
10.1111/ejh.13827
Keywords
MTD (maximal tolerated dose); fetal hemoglobin; hydroxyurea implementation; longitudinal data; pediatric sickle cell
Abstract
Hydroxyurea (HU) has proven benefit in sickle cell anemia (SCA), but HU is still underutilized. The Pediatric Sickle Cell Program of Northern Virginia prescribes HU regardless of symptoms to all SCA patients age ≥ 9 months and prospectively tracks outcomes. HU is dosed to maximum tolerated dosing (MTD), targeting 30% Hgb F. Longitudinal data from 2009-2019 encompassing 1222 HU-eligible and 950 HU-exposure patient-years were analyzed in 2-year intervals for hemoglobin (Hgb), fetal hemoglobin (Hgb F), hospitalizations, transfusions, and treat-and-release ED visits. Comparing HU-eligible patients in the interval prior to HU implementation (2009-2011) to the last interval analyzed after HU implementation (2017-2019), HU usage increased from 33% to 93%, average Hgb increased from 8.3±0.98 to 9.8±1.3 g/dL (p<0.0001), average Hgb F rose from 13±8.7% to 26±9.9% (p<0.0001), hospitalizations decreased from 0.71 (95% CI 0.54-0.91) to 0.2 (95% CI 0.13-0.28) admissions/person-year, sporadic transfusions decreased from 0.4 (95% CI 0.27-0.55) to 0.05 (95% CI 0.02-0.12) transfusions/person-year. Treat-and-release ED visit rates remained unchanged, varying between 0.49 (95% CI 0.36-0.64) and 0.64 (95% CI 0.48-0.83) visits/person-year. By the last interval, 72% of patients had Hgb ≥ 9g/dL, 42% had Hgb F ≥ 30%, 79% experienced no hospitalizations, and 94% received no transfusions. Uniform HU prescription for SCA patients with close monitoring to achieve high Hgb F resulted in significant improvements in laboratory and clinical outcomes within 2 years, which continued to improve over the next 6 years. Rigorous HU implementation in a pediatric sickle cell population is feasible, effective, and sustainable. This article is protected by copyright. All rights reserved.
APA Citation
Phan, Vivian; Park, Ju Ae; Dulman, Robin; Lewis, Angela; Briere, Noravy; Notarangelo, Bailey; and Yang, Elizabeth, "Ten-year Longitudinal Analysis of Hydroxyurea Implementation in a Pediatric Sickle Cell Program" (2022). GW Authored Works. Paper 1330.
https://hsrc.himmelfarb.gwu.edu/gwhpubs/1330
Department
Pediatrics