The right patient, the right treatment: tailoring sickle cell disease care.

Abstract

Sickle cell disease (SCD) remains the most common monogenic disorder worldwide, yet more than a century after its formal discovery, our treatment options and approach to care remain underdeveloped. Despite scientific progress, care for SCD continues to lag behind other chronic conditions, in part due to a lack of consensus on straightforward medical decision-making-driven by a persistent paucity of data and compounded by historical neglect, systemic inequities, and clinical heterogeneity. To truly serve this vulnerable population, we must move toward a more comprehensive, personalized approach to adult care-one that mirrors the individualized pain management plans routinely used in acute care settings. While much of the foundational work in SCD has been in pediatrics, this manuscript focuses on the adult population, where the burden of disease increases and access to specialized care declines. We emphasize the importance of considering genotype, hematologic profile, comorbid conditions, psychosocial context, and health literacy when developing treatment plans in shared decision-making with our patients. Ultimately, we hope this manuscript provides practical insights to help clinicians answer the daily complex questions surrounding sickle cell care: whom to treat and which interventions offer the most meaningful benefit for that particular patient.