Stem cell transplantation found to be more cost-effective than gene therapy for sickle cell disease

by American Society of Hematology

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Stem cell transplantation is the most cost-effective option for long-term care of sickle cell disease in adults when compared with gene therapy and standard of care treatment, according to new research published in Blood .

The analysis is the first to directly compare the value of all three treatment options, finding that the cost of gene therapy needs to be 66% to 71% lower than previously determined to have a competitive value for price.

"Gene therapy is an incredible immune innovation, but it comes with an astronomical cost," said lead study author George Goshua, MD, SM, FACP, assistant professor of medicine in the section of medical oncology and hematology at Yale School of Medicine and Yale Cancer Center, and principal investigator at the Goshua Lab.

"Recent prospective studies suggest that stem cell transplantation is now safer and more efficacious than before for people living with sickle cell disease, but data on its cost-effectiveness, especially in the era of gene therapy, have been limited."

The burden and biology of sickle cell

Sickle cell disease is the most common inherited blood disorder, affecting nearly 8 million people worldwide and approximately 100,000 individuals in the United States. It is more prevalent in people with ancestral origins in regions where malaria is common, including sub-Saharan Africa, the Indian subcontinent, the Arabian Peninsula, and parts of the Mediterranean.

The disorder is characterized by abnormally shaped blood cells that can become lodged in the blood vessels, blocking blood flow and leading to organ damage, infection, and episodes of severe pain.

Current care and curative options

Standard of care management for the disease includes hydroxyurea, pain management, and blood transfusions, but individuals can also achieve lifelong remission through stem cell transplantation or gene therapy, both of which work to replace defective blood-forming stem cells with healthy ones capable of producing normal, non-sickled red blood cells.

Non-myeloablative haploidentical allogeneic stem cell transplantation (NMAC-HID allo-HSCT) uses reduced-intensity conditioning, involving less chemotherapy and radiation to prepare the bone marrow, making it less toxic than traditional transplant and better for older or medically frail patients. It also requires only a half-matched donor instead of a full match, significantly expanding access for individuals living with sickle cell disease, who often have fewer matched donors available.

This type of transplant still carries the risk of graft-versus-host disease (GVHD)—a serious complication in which the immune system attacks donor cells—but at a lower rate than previously observed.

Gene therapy, approved in 2023 by the U.S. Food and Drug Administration for patients 12 years and older with sickle cell disease, uses intensive conditioning followed by an infusion of a patient's own genetically modified and normally functioning stem cells.

Balancing patient choices and policy needs

"From a patient perspective, there are multiple trade-offs across sickle cell treatment options, including differences in eligibility, timing, and a patient's individual values and preferences," said Dr. Goshua.

"From a policy perspective, understanding the cost-effectiveness of all these available treatments can equip governments to make informed, strategic decisions about how to invest in and support the health of their populations living with sickle cell disease.

"That said, it is important for individuals living with sickle cell disease to have access to as many treatments as possible; gene therapy may be the best option for many patients, and these data should not be interpreted as a reason to deny them coverage. Treating physicians must continue to discuss all options with patients in the context of shared patient-physician decision making."

Inside the cost-effectiveness analysis

In the study, the researchers used a decision-analytic model to compare the cost-effectiveness of gene therapy, NMAC-HID allo-HSCT, and standard of care for patients with sickle cell disease aged 18 years or more. They projected outcomes over a patient's lifetime across all accepted willingness-to-pay thresholds (WTP, the maximum amount a consumer or health system is willing to pay per year of optimized health).

To assess the cost-effectiveness of each treatment modality, the researchers determined quality-adjusted life-years (QALYs, how long and how well patients live) and net monetary benefit (NMB, a measure that combines costs and health benefits into a single value). In areas of uncertainty, the researchers relied on the best available clinical data and previously published cost scaling methods.

Key findings on value and outcomes

In the base case, which accounted for key adverse events like GVHD and treatment failure, for adult patients receiving treatment in the United States:

• Gene therapy accrued 22.1 QALYs at a cost of $2.75 million

• NMAC-HID allo-HSCT accrued 20.1 QALYs at a cost of $1.15 million

• Standard of care accrued 14.3 QALYs at a cost of $1.22 million

Additionally, the incremental NMBs for NMAC-HID allo-HSCT were $657,000 and $1,403,000 when compared to standard of care and gene therapy, respectively.

The researchers based the NMAC-HID allo-HSCT model of the study on results of the Phase II trials of the Blood and Marrow Transplant Clinical Trials Network (BMT CTN 1507) and of the Vanderbilt Global Haploidentical BMT Learning Collaborative (VGHLC).

Price thresholds and global implications

The researchers determined that, in the United States, the threshold price for gene therapy, when compared to NMAC-HID allo-HSCT, is $627,000 or $740,000, based on VGHLC and BMT CTN 1507, respectively—a 66% to 71% reduction from gene therapy's current price and a lower price than the $1.3 million cost suggested by the Institute for Clinical Economic Review.

"No matter how much we adjusted from base case assumptions or accounted for uncertainty, NMAC-HID allo-HSCT delivered the best clinical value for cost," said Dr. Goshua.

"Globally, the cost and access to stem cell transplantations are substantial barriers for many. The price thresholds suggested in our U.S. and expanded international analyses may help guide local governmental investment for both curative therapies in sickle cell disease."

Given the nature of the analysis, it had several limitations, including a lack of data on long-term efficacy of both NMAC-HID allo-HSCT and gene therapy, as well as an absence of patient-level data across trials. Additionally, the study used the publicly available list price for gene therapy as negotiated, potentially discounted prices through insurance are not publicly available.

Publication details

Blood (2026) 10.1182/blood.2025032290

Journal information: Blood

Key medical concepts

Sickle Cell DiseaseHematopoietic Stem Cell Transplantation

Clinical categories

Oncology Provided by American Society of Hematology Who's behind this story?

Sadie Harley

BSc Life Sciences & Ecology. Microbiology lab background with pharmaceutical news experience in oil, gas, and renewable industries. Full profile →

Robert Egan

Bachelor's in mathematical biology, Master's in creative writing. Well-traveled with unique perspectives on science and language. Full profile →

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