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

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.