Hydroxyurea to lower transcranial Doppler velocities and prevent primary stroke: the Uganda NOHARM sickle cell anemia cohort

Robert Opoka, Heather A. Hume, Teresa S. Latham, Adam Lane, Olatundun Williams, Jennifer Tymon, Maria Nakafeero, Phillip Kasirye, Christopher M. Ndugwa, Chandy C. John, Russell E. Ware

Research output: Other contribution


In summary,

the NOHARM trial provides additional evidence for the safety and efficacy of short-term hydroxyurea treatment for SCA in sub-Saharan Africa within a clinical trial setting. Data from both the blinded treatment phase and the open-label treatment phase demonstrate clinical and hematological benefits of hydroxyurea in a malarial endemic region, including evidence of reduced TCD velocities and primary stroke prevention. Additional TCD screening programs and clinical trials are urgently needed in sub-Saharan Africa to document the feasibility and benefits of hydroxyurea treatment for children with conditional or even abnormal TCD velocities, given the challenges of providing chronic blood transfusions. To help prevent primary stroke in this high-risk population, future studies should also focus on optimal dosing strategies and monitoring regimens, in an effort to determine the overall feasibility and safety of introducing hydroxyurea therapy for SCA widely across sub-Saharan Africa.

Original languageUndefined/Unknown
Publication statusPublished - 1 Jun 2020

Publication series

NamePaediatrics and Child Health, East Africa

Cite this