Evaluating the long-term benefits of hydroxyurea in pediatric sickle cell anemia.

ABSTRACT:

Abstract

Hydroxyurea is the primary disease-modifying medication for sickle cell anemia (SCA), but its long-term effects, particularly how these effects change over time, are not well understood. This study aimed to quantify the effects of hydroxyurea on clinical and laboratory outcomes in children with SCA over a prolonged period of use. We conducted a quasi-experimental study using contemporary difference-in-differences and dynamic event study analyses on a longitudinal cohort of 2147 children with SCA (hemoglobin SS or hemoglobin SSβ0, HbSS/HbSβ0) from 2010 to 2021. The primary outcomes included emergency department (ED) visits per year, hospital days per year, and annual average hemoglobin concentration. Hydroxyurea use was associated with fewer ED visits per year (average trea

SUBMITTER: George P 

PROVIDER: S-EPMC12281001 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature