Children With Sickle Cell Anemia Who Receive Hydroxyurea Have Less Transfusion Need

Hydroxyurea therapy, when escalated to the maximum tolerated dose (MTD), clearly reduces the need for blood transfusions among children with sickle cell anemia (SCA). This is according to an analysis recently published in American Journal of Hematology of data from the longitudinal phase I/II REACH trial.

As described by the trial authors, SCA is associated with life-threatening complications that strain the transfusion resources of health care systems globally. This is especially the case for African nations such as those where REACH was performed. Based on the trial’s results, however, lead author Alexandra Power-Hays, MD, of Cincinnati Children’s Hospital Medical Center in Ohio, proposes that “universal testing and hydroxyurea treatment at MTD could potentially prevent 21% of all pediatric transfusions administered in sub-Saharan Africa.”

The REACH trial sought to compare the transfusion utilization of children with SCA before and while receiving a hydroxyurea regimen. The regimen in the trial was structured as that for the first six months hydroxyurea was administered as a fixed dose (15–20 mg/kg/day) and then escalated over a course of 18 months up to MTD.

The trial enrolled 635 children from Angola, Democratic Republic of Congo, Kenya, and Uganda, collecting 4,124 person-years of observation. Out of the total enrolled study group, 258 children (40.4% of participants) received transfusions over the course of the study, with 545 transfusions among them.

According to incidence rate ratios (IRR) calculated by the researchers, transfusions at MTD were reduced by 75% as compared against pretreatment (IRR 0.25, 95% CI, 0.18-0.35, P< .0001) and by 50% as compared against the fixed-dose period (IRR 0.50, 95% CI, 0.39-0.63, P< .0001). The transfusion rate per 100 person-years also showed improvement, having been calculated as 43.2 before treatment and falling to 21.7 while on fixed dose, then 14.5 during dose escalation, and finally 10.8 while on MTD.

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;99(4):625-632. doi:10.1002/ajh.27244