Hydroxyurea Reduces Transfusion Requirements in Children With Sickle Cell Anemia

Hydroxyurea treatment reduced the incidence of blood transfusion by 75% among pediatric patients in sub-Saharan Africa with sickle cell anemia, according to the phase I/II REACH trial.

The study was led by Alexandra Power-Hays, MD, of the Cincinnati Children’s Hospital Medical Center, and published in the American Journal of Hematology.

Dr. Power-Hays and colleagues evaluated transfusion utilization in children with sickle cell anemia before and during hydroxyurea treatment. Risk factors for higher transfusion rates include younger age at enrollment, lower hemoglobin, and palpable splenomegaly.

Hydroxyurea Significantly Reduces Transfusions in Sickle Cell Disease

Following a pretreatment screening period of two months, patients received six months of hydroxyurea at a fixed dose of 15 to 20 mg/kg/day, 18 months of dose escalation, then stable dosing at the maximum tolerated dose (MTD).

A total of 545 transfusions were administered to 258 of 635 (40.4%) patients, and 133 (20.9%) patients received two or more transfusions. The transfusion rate per 100 person-years was 43.2 before hydroxyurea, 21.7 on fixed dose, 14.5 during dose escalation, and 10.8 on MTD.

MTD treatment reduced transfusion rate by 75% compared with the pretreatment period (IRR 0.25, 95% confidence interval [CI] 0.18–0.35, P<0.0001), and by 50% compared with fixed dose treatment (IRR 0.50, 95% CI 0.39–0.63, P<0.0001).

“If widely implemented, universal testing and hydroxyurea treatment at MTD could potentially prevent 21% of all pediatric transfusions administered in sub-Saharan Africa,” wrote Dr. Power-Hays and colleagues.

Recurrent transfusions can contribute to iron overload, erythrocyte alloimmunization, and non-immune hemolytic transfusion reactions, according to the authors. “Increasing hydroxyurea access for [sickle cell anemia] should decrease the transfusion burden and increase the overall blood supply,” they concluded.

Related: Could Isoquercetin Solve Thromboinflammation in Sickle Cell Disease?

Reference

Power-Hays A, Tomlinson GA, Tshilolo L, et al. Reducing transfusion utilization for children with sickle cell anemia in sub-Saharan Africa with hydroxyurea: analysis from the phase I/II REACH trial. Am J Hematol. 2024. doi.org/10.1002/ajh.27244