Clinical consultation: The pharmacist's role in hydroxyurea management for sickle cell disease

Sickle cell disease (SCD) is a genetic disorder caused by a mutation in the β-globin gene, leading to the production of abnormal sickle hemoglobin (HbS). This clinical consultation explores the pivotal role of hydroxyurea as a disease-modifying therapy in SCD and examines how pharmacist-led interventions can optimize its use to improve patient outcomes.

Hydroxyurea increases fetal hemoglobin (HbF) production, reducing HbS polymerization and lowering the frequency of vaso-occlusive crises, acute chest syndrome, and other complications. Despite its proven efficacy, barriers such as poor adherence, inadequate patient education, and suboptimal dosing hinder its effective use. Pharmacists, as integral members of the multidisciplinary care team, are ideally positioned to address these challenges. By providing patient education, regular laboratory monitoring, and dose titration to the maximum tolerated dose (MTD), pharmacists can enhance hydroxyurea's therapeutic benefits. Studies have shown that pharmacist-managed protocols significantly improve adherence, increase the proportion of patients achieving MTD, and lead to better clinical outcomes, such as higher HbF levels and fewer hospitalizations. Although emerging treatments such as gene therapies offer promise, hydroxyurea remains the most accessible and effective option for many patients.

Pharmacists play a crucial role in optimizing hydroxyurea therapy for patients with SCD by improving adherence and maximizing dosing strategies. Their involvement ensures that more patients experience the full therapeutic benefits of hydroxyurea, contributing to better long-term outcomes. Expanding pharmacist engagement in SCD management will continue to be essential, particularly as new treatments are developed.