Intermittent or uneven daily administration of low‐dose hydroxyurea is effective in treating children with sickle cell anemia in Angola

摘要

Abstract Background Although hydroxyurea is proven effective in treatment of sickle cell anemia (SCA) and is widely prescribed in high‐income countries, due to questions about feasibility of treating large numbers of patients in resource‐limited health systems, its use is limited in sub‐Saharan Africa (SSA), where most children with SCA live. We assessed hematological response and toxicity of hydroxyurea treatment for SCA in Angola. Methods Retrospective study of children with SCA (not selected for clinical severity) treated on a fixed dose of hydroxyurea for at least 6?months. Because only the 500?mg capsule was available, dose was averaged weekly. We evaluated toxicity events and magnitude of hydroxyurea‐induced changes in blood counts and compared patients who received a uniform daily dose to those prescribed intermittent or uneven daily doses. Results Only 13% of 303 patients received a uniform dose of hydroxyurea daily. Dose ranged from 16.5 to 22.8?mg/kg/day. Hydroxyurea increased HGB and mean cell volume values by 0.5?g/dL ( P ??0.0001) and 8?fL ( P ??0.0001), while ANC, PLT, and ARC decreased 1.1?×?10 9 /L ( P ??0.0001), 34?×?10 9 /L ( P ?=??0.0001), and 19?×?10 9 /L ( P ?=?0.0008), respectively. There were no differences in magnitude of hydroxyurea‐induced changes between patients prescribed intermittent or uneven doses and uniform daily doses, or between those treated in the lower and higher dose quartiles. Hematological toxicity events were mild and reversible. Conclusion Intermittent or uneven daily dosing of hydroxyurea is as effective as fixed daily doses in treating SCA. This strategy may enable treatment of additional children with SCA in SSA.