Alternative dosing and prevention of transfusions (adapt): a prospective trial evaluating transfusion utilization and the feasibility of pharmacokinetic hydroxyurea

: Blood transfusions are a scarce resource globally and particularly in sub-Saharan Africa. Without disease-modifying therapy, in sub-Saharan Africa sickle cell anemia (SCA) has excess morbidity, mortality, and healthcare resource utilization, specifically requiring a high reliance on blood transfusions. Hydroxyurea is a safe medication for the treatment of SCA that can decrease rates of blood transfusions. The widespread use of hydroxyurea in sub-Saharan Africa is limited in part, however, by logistical challenges in determining an individual patient’s optimal dose. Pharmacokinetic (PK) dosing of hydroxyurea may streamline the dosing process, maximizing the clinical benefits and minimizing toxicities, while reducing the time and resources needed to determine the optimal dose.