Lesser-known medication safety considerations in sickle cell disease.

INTRODUCTION: Sickle cell disease (SCD) is a clinically heterogeneous condition in which individuals experience markedly different disease trajectories. As more people with SCD now survive to adulthood, their disease becomes more complicated due to co-morbidities both related and not related to SCD. Thus, a precision-oriented approach is essential when initiating new treatments to ensure the delivery of meaningful and sustained benefits for people with SCD. AREAS COVERED: This expert review examines selected, underrecognized safety concerns related to disease-modifying therapies for SCD as well as for other medications often used in this population. We discuss examples of safety issues involving SCD-modifying therapies, cardiovascular and renal medications, antibiotics, and corticosteroids, highlighting how the unique pathophysiology of SCD may influence drug safety, tolerability, and organ injury. The review is based on a narrative assessment of published clinical trials, observational studies, post-marketing safety reports, registry data, and expert clinical experience. EXPERT OPINION: Although SCD is recognized as a highly heterogeneous condition, this issue is not consistently considered in clinical trial design or when using non-SCD therapies in this population. The recent emergence of new drugs emphasizes the importance of longitudinal monitoring, real-world safety assessment, and multidisciplinary specialist care.