Hydroxyurea as Treatment for Children With Sickle Cell Anemia in Sub-Saharan Africa
The efficacy, safety, and feasibility of using hydroxyurea as a therapeutic agent for sickle cell anemia have not been assessed in many populations.
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While hydroxyurea is an effective therapy for patients with sickle cell anemia, its usage has yet to be assessed for many populations. A prospective study was recently conducted to determine the feasibility, efficacy, and safety of using hydroxyurea in a population of children in sub-Saharan Africa, and the results were presented at the 60th American Society of Hematology (ASH) Annual Meeting in San Diego, California.
The study authors noted that most research on the use of hydroxyurea has occurred in the United States and Europe, and development of treatment standards in other regions might need to account for co-occurring conditions that patients may experience in localities with fewer resources and differing infections.
The researchers treated 606 children from 1 to 10 years of age (median age 5.4 years at enrollment) with an average hydroxyurea starting dose of 17.5 ± 1.8 mg/kg, daily, at 4 centers located in Angola, Democratic Republic of the Congo, Kenya, and Uganda. Dosage increases began at 6 months and continued until evidence of hematologic toxicity.
Study retention and adherence were high, and a maximum tolerated dose was reached by 85% of patients (average maximum tolerated dose 22.5 ± 4.9 mg/kg/day). Within the initial 3 months of the study, dose-limiting toxicity was experienced by 4.9% of the patients. The treatment was generally well tolerated with low occurrences of toxicities.
Hydroxyurea treatment resulted in clinically significant improvements in multiple hematological parameters and was associated with lower levels of transfusions, vaso-occlusive pain, and acute chest syndrome. The rate and severity of malaria were also reduced, and all-cause mortality dropped from 3.6 to 1.1 deaths per 100 patient years.
Hydroxyurea showed good feasibility and efficacy as a treatment for sickle cell anemia and its complications in this patient population and it had a good safety profile.
Disclosures: One of the study authors declared multiple affiliations with pharmaceutical industry. For the full list of disclosures, please refer to the original study.
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1. Tshilolo L, Tomlinson G, Williams TN, et al. Realizing effectiveness across continents with hydroxyurea (REACH): a prospective multi-national trial of hydroxyurea for sickle cell anemia in sub-Saharan Africa. Oral presentation at: 60th ASH Annual Meeting and Exposition; December 1-4, 2018; San Diego, CA.