Bone marrow transplantation for sickle cell disease. The European experience.

PURPOSE: In Belgium and France, 42 patients underwent bone marrow transplantation (BMT) for treatment of sickle cell disease. PATIENTS AND METHODS: The patients were young and symptomatic, but without chronic organ damage. Engraftment occurred in all patients and was sustained in 36. These 36 patients became free of symptoms and had a change in electrophoresis of their hemoglobin toward the donor's pattern. RESULTS: In five patients, engraftment was followed by bone marrow rejection. Two of these five patients underwent a second transplant, one at 62 days and the other at 21 months after the first transplant, and they are both doing well. The other three patients had autologous recovery of their own bone marrow. One patient died 3 months after marrow transplant of complications of graft-versus-host disease (GVHD). All the other patients are alive, with follow-up ranging from 1 to 75 months. CONCLUSIONS: Concerning the long-term side effects, six patients had chronic GVHD disease. So far, eight patients returned to Africa where they are continuing to do well.