Allogeneic bone-marrow transplantation in infantile malignant osteopetrosis.
Two infants with malignant osteopetrosis were treated by allogeneic bone-marrow transplantation after marrow ablation with busulphan and cyclophosphamide. Engraftment without graft-vs-host disease occurred in both cases. The first child established stable chimerism and is clinically well 30 months after receiving a histocompatible sibling bone-marrow graft: haematological, radiological, and biochemical features of osteopetrosis have completely resolved. The second child was non-identical with the marrow donor (his older sister) at a single HLA-B locus and received cyclosporin A as prophylaxis for graft-vs-host disease. 11 months after transplantation haematological abnormalities have resolved and radiological features have improved. The findings indicate that allogeneic bone-marrow transplantation should be considered the treatment of choice for infants with severe osteopetrosis who have histocompatible siblings.
D001788:Blood Grouping and Crossmatching; D016026:Bone Marrow Transplantation; D003524:Cyclosporins / Q000008:administration & dosage; D005500:Follow-Up Studies; D005542:Forearm / Q000000981:diagnostic imaging; D006680:HLA Antigens / Q000276:immunology; D000949:Histocompatibility Antigens Class II / Q000276:immunology; D006801:Humans; D007223:Infant; D008297:Male; D010022:Osteopetrosis / Q000151:congenital / Q000628:therapy*; D011859:Radiography; D014184:Transplantation, Homologous
D003524:Cyclosporins; D006680:HLA Antigens; D000949:Histocompatibility Antigens Class II