Bone marrow transplantation for severe sickle cell anaemia

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Abstract
Summary. Five children with sickle cell anaemia underwent bone marrow tranplantation (BMT) for severe clinical disease. The conditioning regimen for BMT was in busulfan plus cyclophosphamide. The allograft contained more than 5.108 nucleated cells per kg recipient. Prophylaxis of GVHD consisted of methotrexate and cyclosporin A. Therapy was well tolerated. Duration of neutropenia (50 × 109/1) occurred between day 12 and 45. The patients have been folowed up for 8–28 months. No major infections occurred and long‐term BMT‐related toxicity was limited to mild, chronic GVHD in one patient. Mean haemoglobin levels remained above 10 g/dl. Haemoglobin electrophoresis showed AS patterns in all grafted patients—all marrow donors having sickle cell trait. From our preliminary data, we conclude that BMT or sickle cell anaemia is curative, well tolerated and should be proposed for suitable patients.