Optimization of hydroxyurea in sickle cell disease in Brazil

Abstract

Despite sickle cell disease (SCD) being a well-recognized and highly prevalent condition identified early through neonatal screening programs, it represents a substantial public health challenge due to high morbidity and premature mortality rates. Hydroxyurea (HU) is the only available disease-modifying therapy for SCD approved in Brazil. Indeed, its underutilization highlights the need for improved therapeutic strategies to enhance adherence and management of SCD. Innovative formulations of HU might favor treatment adherence and precise dosing. Thus, we aimed to describe HU's pharmacological characteristics, clinical efficacy, and tolerability, including dose escalation. Recent interventional and observational studies revealed the efficacy and safety of an innovative formulation: dispersible scored tablets of 100 mg and 1000 mg, allowing easier dose adjustments and, consequently, more precise dosing. The 100 mg tablets scored can be cut into two parts of 50 mg, and the 1000 mg tablets can be cut into four parts of 250 mg. The fractionating dose is possible due to the formulation technology that allows the tablet to be cut with a uniform amount of drug in each part. This new formulation of HU, suitable for children, may influence the prognosis of SDC, regardless of associated symptoms.