Successful matched-unrelated bone marrow transplantation in a patient with beta-thalassemia major.
The authors describe a 5-year-old boy with beta-thalassemia major who received bone marrow transplantation (BMT) from a human leukocyte antigen (HLA)-matched unrelated donor. The conditioning regimen consisted of 16 mg/kg busulfan and 200 mg/kg cyclophosphamide. The transplantation was complicated with grade II graft-versus-host disease, although prophylaxis with cyclosporine and short-term methotrexate was carried out. Cytomegalovirus disease occurred at 2 months after transplantation but was controlled successfully. The child remains disease-free and in good clinical condition 53 months after BMT. The authors suggest that BMT from an HLA-matched unrelated donor could be considered as an alternative treatment in patients with beta-thalassemia major when no HLA-matched donor is available.
D000328:Adult; D001853:Bone Marrow / Q000276:immunology / Q000378:metabolism; D016026:Bone Marrow Transplantation / Q000276:immunology*; D002675:Child, Preschool; D005260:Female; D005838:Genotype; D006680:HLA Antigens / Q000235:genetics / Q000276:immunology; D006801:Humans; D008297:Male; D014019:Tissue Donors; D017086:beta-Thalassemia / Q000276:immunology* / Q000628:therapy*
D006680:HLA Antigens