Lesser-known medication safety considerations in sickle cell disease

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.

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.

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.