Expanding Bone Marrow Transplantation for Sickle Cell Disease?

Periodic blood transfusions and hydroxyurea have improved quality of life for some patients with sickle cell disease (SCD). But in many others who continue to do poorly, recurrent pain crises, infections, acute chest syndrome, and strokes result in frequent hospitalizations and shortened life expectancy. Allogeneic bone marrow transplantation has the potential to cure SCD, but the procedure has been used infrequently because of the difficulty in locating human leukocyte antigen (HLA)–matched donors, concerns about the toxicity of conditioning regimens, and the need for lifelong immunosuppression.

Now, investigators describe durable marrow engraftment in 11 of 17 SCD patients using nonmyeloablative conditioning with antithymocyte globulin, fl…