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

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 × 109 /L (P < 0.0001), 34 × 109 /L (P = < 0.0001), and 19 × 109 /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.

A Paper-Based Test for Screening Newborns for Sickle Cell Disease.

The high cost, complexity and reliance on electricity, specialized equipment and supplies associated with conventional diagnostic methods limit the scope and sustainability of newborn screening for sickle cell disease (SCD) in sub-Saharan Africa and other resource-limited areas worldwide. Here we describe the development of a simple, low-cost, rapid, equipment- and electricity-free paper-based test capable of detecting sickle hemoglobin (HbS) in newborn blood samples with a limit of detection of 2% HbS. We validated this newborn paper-based test in a cohort of 159 newborns at an obstetric hospital in Cabinda, Angola. Newborn screening results using the paper-based test were compared to conventional isoelectric focusing (IEF). The test detected the presence of HbS with 81.8% sensitivity and 83.3% specificity, and identified SCD newborns with 100.0% sensitivity and 70.7% specificity. The use of the paper-based test in a two-stage newborn screening process could have excluded about 70% of all newborns from expensive confirmatory testing by IEF, without missing any of the SCD newborns in the studied cohort. This study demonstrates the potential utility of the newborn paper-based test for reducing the overall cost of screening newborns for SCD and thus increasing the practicality of universal newborn SCD screening programs in resource-limited settings.