Bone marrow transplantation in Fanconi anaemia.
Five patients with Fanconi anaemia have been treated by bone marrow transplantation from HLA identical donors. Only one patient survived for more than 3 years. She is now perfectly healthy with complete haematological reconstitution with chimaerism and disparition of chromosomal abnormalities. In contrast, four patients died of acute severe GVHD soon after grafting. In addition, all had signs of severe cyclophosphamide toxicity. This evolution could be explained by a special sensitivity of FA cells to alkylating agents and may indicate the need to modify the conditioning regimen in FA patients.
D000741:Anemia, Aplastic / Q000628:therapy*; D016026:Bone Marrow Transplantation; D002648:Child; D002869:Chromosome Aberrations; D025063:Chromosome Disorders; D003520:Cyclophosphamide / Q000009:adverse effects; D005199:Fanconi Anemia / Q000628:therapy*; D005260:Female; D006087:Graft vs Host Reaction; D006680:HLA Antigens; D006801:Humans; D008214:Lymphocytes / Q000648:ultrastructure; D008297:Male; D014185:Transplantation, Isogeneic
D006680:HLA Antigens; D003520:Cyclophosphamide