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Ten-year longitudinal analysis of hydroxyurea implementation in a pediatric sickle cell program

Vivian Phan1, Ju Ae Park2, Robin Dulman1

  • 1Pediatric Specialists of Virginia, Fairfax, Virginia, USA.

European Journal of Haematology
|July 11, 2022
PubMed

Insights

Hydroxyurea (HU) significantly improves sickle cell anemia (SCA) outcomes by increasing hemoglobin and fetal hemoglobin levels. This treatment is feasible, effective, and sustainable for pediatric SCA patients, reducing hospitalizations and transfusions.

Area of Science:

  • Hematology
  • Pediatric Medicine
  • Pharmacology

Background:

  • Sickle cell anemia (SCA) is a debilitating genetic blood disorder.
  • Hydroxyurea (HU) is a proven treatment for SCA but remains underutilized.
  • Effective implementation strategies for HU in pediatric SCA are needed.

Purpose of the Study:

  • To evaluate the impact of a uniform hydroxyurea (HU) prescription protocol on clinical and laboratory outcomes in pediatric patients with sickle cell anemia (SCA).
  • To assess the feasibility, effectiveness, and sustainability of HU treatment in a real-world pediatric setting.

Main Methods:

  • A prospective, longitudinal study analyzed data from 2009-2019 for 1222 HU-eligible pediatric SCA patients.
  • Hydroxyurea was prescribed regardless of symptoms to all patients aged ≥9 months, dosed to maximum tolerated dosing (MTD) targeting 30% fetal hemoglobin (Hgb F).
  • Outcomes including hemoglobin (Hgb), Hgb F, hospitalizations, transfusions, and emergency department (ED) visits were tracked in 2-year intervals.

Main Results:

  • Hydroxyurea usage increased from 33% to 93% from 2009-2011 to 2017-2019.
  • Average Hgb increased from 8.3 to 9.8 g/dL (p<0.0001), and average Hgb F rose from 13% to 26% (p<0.0001).
  • Hospitalizations decreased from 0.71 to 0.2 admissions/person-year, and transfusions decreased from 0.4 to 0.05 transfusions/person-year. Treat-and-release ED visits remained unchanged.

Conclusions:

  • Uniform hydroxyurea (HU) prescription and close monitoring in pediatric sickle cell anemia (SCA) patients lead to significant improvements in laboratory and clinical outcomes.
  • These improvements are achievable within 2 years and are sustainable over longer periods.
  • Rigorous implementation of HU is feasible and effective in a pediatric SCA population, reducing disease burden.

Keywords:
MTD (maximal-tolerated dose)fetal hemoglobinhydroxyurea implementationlongitudinal datapediatric sickle cell

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