Hydroxyurea for Secondary Stroke Prevention in Children with Sickle Cell Anemia in Nigeria:a Randomized Controlled Trial
AUTHORS
- PMID: 36322937 [PubMed].
ABSTRACT
We tested the hypothesis that fixed oral moderate-dose hydroxyurea (20 mg/kg/day) for initial treatment of secondary stroke prevention results in an 80% relative risk reduction of stroke or death when compared to fixed oral low-dose hydroxyurea (10 mg/kg/day) in a phase III, double-blind, parallel-group, randomized controlled trial in children with sickle cell anemia (SCA) living in Nigeria. The median participant follow-up was 1.6 years (interquartile range 1.0-2.3) with a planned minimum follow-up of 3.0 years. A total of 6 recurrent strokes and 2 deaths versus 5 recurrent strokes and 3 deaths occurred in the low- and moderate-dose groups, respectively. The incidence rate ratio (IRR) of the primary outcome measure of stroke or death in the low- and moderate-dose hydroxyurea treatment groups was 0.98 (95% CI 0.32 – 3.00), P=0.97. The trial was stopped early due to no clinical difference in the incidence rates of the primary outcome measure. The incidence rates of recurrent strokes were 7.1 and 6.0 per 100 person-years in the low- and moderate-dose groups, respectively, IRR= 1.18 (95% CI: 0.30-4.88); P=0.74. As a measure of adherence to the oral hydroxyurea therapy, the median percent of returned pills were 3.0% and 2.6% in the low- and moderate-dose groups, respectively. No participant had hydroxyurea therapy stopped for myelosuppression. For children with SCA in low-income settings, without access to regular blood transfusion therapy, initial low-dose hydroxyurea is a minimum known efficacious dose for secondary stroke prevention. The ID number assigned to the study is NCT02675790.
Tags: faculty publications 2022