Improving Outcomes for Patients With Sickle Cell Disease in the United States: Making the Case for More Resources, Surveillance, and Longitudinal Data

Abstract

Importance: Although considered a rare disease with fewer than 200 000 cases annually in the US, sickle cell disease (SCD) is the most common and clinically significant inherited blood disorder in the US and worldwide. Despite the relatively high prevalence of this rare disease, there is a paucity of longitudinal data available to evaluate access to care or to identify quality metrics.

Observations: This review discusses why systematic data collection for SCD through population-wide surveillance programs can help to facilitate progress in treatment. It also explores the importance of having both a longitudinal clinical registry and a national surveillance program to improve resource utilization, clinical outcomes, and provide an equitable foundation for care.

Conclusions and relevance: Federal funding should be appropriately allocated to establish and maintain a national SCD surveillance system supported by the Centers for Disease Control and Prevention, as well as a longitudinal registry available at recognized sickle cell centers.