Stem Cell Transplantation Found More Cost-Effective Than Gene Therapy for Sickle Cell Disease Patients

A new study published in the journal Blood has found that stem cell transplantation is the most cost-effective long-term treatment option for adults with Sickle Cell Disease when compared with gene therapy and standard care.

Researchers led by George Goshua compared the costs and effectiveness of three treatment approaches. While gene therapy was associated with the greatest gains in quality of life and life expectancy, its exceptionally high cost reduced its overall economic value. According to the analysis, the price of gene therapy would need to decrease by 66% to 71% to become economically competitive with stem cell transplantation.

Sickle cell disease is the most common inherited blood disorder worldwide, affecting approximately 8 million people. The disease causes red blood cells to become abnormally shaped, which can block blood vessels and lead to organ damage, infections, and severe pain episodes.

The study evaluated non-myeloablative haploidentical allogeneic stem cell transplantation, a procedure that requires less chemotherapy and radiation than traditional transplants. This approach is considered less toxic and may be more suitable for older patients or those with complex medical conditions.

The findings showed that gene therapy provides an average of 22.1 quality-adjusted life years (QALYs) at a cost of $2.75 million, whereas stem cell transplantation provides 20.1 QALYs at a cost of $1.15 million. Standard care resulted in 14.3 QALYs at a cost of $1.22 million.

The researchers emphasized that despite the economic advantage of stem cell transplantation, gene therapy may still be the best option for certain patients. They cautioned that these findings should not be used to restrict patient access to gene therapy. Instead, treatment decisions should be based on individual patient circumstances and made through shared decision-making between patients and physicians.

The study also acknowledged several limitations, including the lack of long-term data on the effectiveness of both gene therapy and stem cell transplantation.

https://medicalxpress.com/news/2026-06-stem-cell-transplantation-effective-gene.html