Abstract
Background: Very few trials of hydroxyurea efficacy and safety have been conducted in sub-Saharan Africa. We aimed to evaluate the efficacy and safety of hydroxyurea and its utility in low-resource settings. Methods: We conducted a prospective comparative trial in patients with SCA. 128 patients were enrolled and divided into two groups. 68 patients were treated with hydroxyurea at a dose of 10-20 mg/kg/day and 62 patients in a control group without hydroxyurea. The endpoints evaluated were feasibility, safety, and benefit (laboratory variables, sickle cell-related events, transfusions). Results: The patients assigned to hydroxyurea treatment had a lower annual rate of crises than the control group (median 2.9 vs. 5.3 crises per year, p=0.001), a lower annual rate of hospitalizations (median 2.2 vs. 4.7, p=0.002), and a lower annual rate of transfusions (median 1.3 vs. 5.1, p=0.001). We observed a significant increase in Hb F from 11.77% to 14.6% (p=0.001) in patients treated with hydroxyurea. We also observed a significant increase in the mean Hb level from 7.3 g/dL to 9.2 g/dL in patients treated with hydroxyurea (p=0.004). Patients treated with hydroxyurea also have a beneficial effect on WBC and platelet levels by reducing leukocytosis and thrombocytosis. The annual number of infectious complications was significantly lower in the group of patients treated with hydroxyurea. Conclusion: Hydroxyurea has an important clinical benefit by reducing the incidence of vaso-occlusive events, infections, and transfusions, which translates into fewer hospitalizations. The main problem is that it is not accessible to most of our patients who live in poor socioeconomic conditions.