Exploring equitable treatment options for pediatric patients with sickle cell disease

Patients with sickle cell disease (SCD) often grapple with diminished quality of life and a reduced life expectancy. The primary curative treatment, allotransplantation, carries risks, including transplant-related mortality. Gene therapy, if approved for SCD, could potentially provide a lifelong cure without the inherent risks associated with allotransplantation. Addressing the need for equitable treatment options for this historically marginalized pediatric patient population in the United States, researchers from Yale Cancer Center conducted a groundbreaking study to assess the cost-effectiveness of allotransplantation therapy and gene therapy.

Presented at the American Society for Hematology (ASH) Annual Meeting, the study, led by Dr. George Goshua, revealed that allotransplantation had an incremental cost-effectiveness ratio (ICER) of $138,000 per quality-adjusted life-year (QALY), while gene therapy showed an ICER of $253,000. Despite the apparent expense, both therapeutic strategies met distributional equity standards in the United States.

Dr. Goshua, an assistant professor of medicine (Hematology) at Yale School of Medicine, stressed the importance of considering equity in decision-making for pediatric SCD treatment. The researchers aim to contribute transparent benchmarks for stakeholders involved in crucial funding decisions, fostering a more equitable and value-informed approach to pediatric SCD care.