A recent study presented at the 66th American Society of Hematology Annual Meeting reveals a concerning trend in the treatment of sickle cell disease (SCD). Despite the availability of safe and effective disease-modifying therapies (DMTs), only a quarter of patients living with SCD are prescribed these treatments, particularly the newer options.
Dr. Omar Niss, director of classical hematology at Cincinnati Children’s Hospital Medical Center, emphasizes the gravity of this issue. “Underuse of these therapies is a real problem,” he states, highlighting the daily benefits these medications provide in reducing pain and increasing hemoglobin levels, as well as their long-term protective effects on organs and overall well-being.
SCD, affecting approximately 100,000 individuals in the United States and one out of every 365 Black or African American births, is the most common inherited blood disorder. The study, which analyzed data from 22,793 patients across 14 U.S. sites, found that only 24% of patients were prescribed one or more DMT. Even among those with the most severe variant of SCD, only 35% received these therapies.
The research also uncovered geographical disparities in DMT utilization, with the highest rates in the Northeast U.S. (33%) and the lowest in the Southwest (15.4%). While there has been a slight increase in DMT use from 2015 to 2023, rising from 7.2% to 19.8%, this growth is primarily attributed to hydroxyurea, a drug available for over 35 years.
Dr. Niss expresses disappointment in these findings, stating that ideally, all patients with severe forms of SCD should be receiving at least one DMT. He calls for further investigation into the barriers preventing wider use of these therapies, emphasizing that there is no justification for their underutilization given their proven efficacy.
This study underscores the urgent need to address health disparities in SCD treatment, ensuring that all patients have access to these life-improving therapies regardless of their geographic location or disease severity.
See “Disease-Modifying Therapies Remain Underused in Sickle Cell Disease Despite Safety and Efficacy” (December 7, 2024)
