Bone Marrow Transplant: A Cure for Sickle Cell Disease

Bone marrow transplant, also called a stem cell transplant, is the established curative treatment for sickle cell disease. The procedure replaces the patient's bone marrow with healthy stem cells from a donor, ideally a matched sibling, or a matched unrelated or half-matched family donor. The donor cells then produce normal red blood cells instead of sickle-shaped ones.

For patients with severe sickle cell disease, a successful transplant can stop painful crises, prevent further organ damage and significantly improve quality of life. Before the transplant, patients receive chemotherapy to clear the existing marrow, after which the healthy stem cells are infused. The process carries risks such as infection, graft rejection and graft-versus-host disease, which is why careful monitoring in an experienced centre matters.

Transplant is not suitable for every patient. It works best in younger patients with severe disease who have a well-matched donor. Gene therapies, approved in 2023, now offer another potentially curative option that uses the patient's own modified stem cells, although their cost and availability remain very limited.