source: Haematologica

year: 2020

authors: Robert O. 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

In sub-Saharan Africa, sickle cell anemia (SCA) remains a significant public health problem with high mortality: an estimated 50-90% of affected children die before 5 years of age. A high prevalence of stroke is particularly devastating for children and has substantial morbidity. Hydroxyurea is now recommended across the lifespan as a safe and effective disease-modifying therapy for SCA, but is not routinely available in sub-Saharan Africa.

NOHARM (NCT01976416), a randomized double-blinded, placebo-controlled trial, was among the first prospective studies to investigate hydroxyurea treatment for children with SCA living in a malaria endemic region within Africa. The NOHARM trial consisted of a blinded phase (Year 1) and an open-label phase (Year 2). Initially children 1.00-4.99 years of age were randomized either to placebo or fixed-dose hydroxyurea (20 mg/kg/day). The Year 1 results demonstrated that short-term hydroxyurea treatment was both safe and efficacious in this young patient population living in a malarial endemic region. We now analyze Year 2 data from the open-label phase, in which all NOHARM participants received hydroxyurea at 20 mg/kg/day, to compare the effects of hydroxyurea on SCA-related morbidity and also to provide serial transcranial Doppler (TCD) data, which were not included in the original randomized report.

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