Treatments for children with sickle cell

New research reveals equitable treatments for pediatric sickle cell.


People with sickle cell disease (SCD) often have a tough time with their health and may not live as long. The primary treatment, called allotransplantation, can help, but it has some dangers, like the risk of not surviving the transplant. Gene therapy, if it gets approved for SCD, might be a safer way to cure the disease for good without the risks of allotransplantation.

Yale researchers studied the costs of two treatments for sickle cell disease in kids: allotransplantation therapy and gene therapy. They wanted to find fair options for these children who often face challenges. Dr. George Goshua will present the findings at the American Society for Hematology Annual Meeting on December 10.

The study compared the cost-effectiveness of two treatments using an incremental cost-effectiveness ratio (ICER) measure. For allotransplantation, it was $138,000 per quality-adjusted life-year (QALY), and for gene therapy, it was $253,000.

Goshua, an assistant professor of medicine (Hematology) at Yale School of Medicine, said, “While these therapeutic strategies appear to be expensive, and they are, pursuing allotransplantation or gene therapy both meet distributional equity standards in the United States.” 

Researchers stress the need to be fair when deciding how to treat kids with sickle cell disease. They want to offer transparent and fair benchmarks for essential funding decisions. Dr. Goshua hopes more groups will also focus on fairness in their decisions.

In conclusion, the researchers want to treat kids with sickle cell disease fairly. They want to set transparent and fair guidelines for important funding choices. Dr. Goshua also wishes other groups to follow this fairness approach in their decisions.


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