[Bone marrow transplantation in Fanconi's anemia. Results in 5 patients]

Fanconi's anaemia (FA) is a form of congenital bone marrow failure characterised by progressive pancytopenia and course usually fatal. Bone marrow transplantation (BMT) offers a chance to restore haemopoiesis, as in bone marrow aplasia of any other aetiology. Five children (4M, 1F) with FA were subjected to BMT. Their ages ranged between 4 and 13 years. The time elapsed between diagnosis and BMT ranged between 6 months and 10 years. Three patients had been previously treated with prednisolone and oxymetholone. The donor age ranged from 20 months to 25 years. The correlation donor sex/recipient sex was female/male in 4 cases (siblings, 3; mother, 1) and male/female in the remainder (one brother). All the siblings involved were HLA-identical, with a negative mixed lymphocyte culture; the mother shared 5 antigens with a different DR. The conditioning regimen was that proposed by Gluckman. BMT was performed by infusion of 2.2 to 5.3 x 10(8) bone marrow cells per kg. Cyclosporin A was used in the prophylaxis of graft versus cells host disease (GVHD), associated to methotrexate in one case and to prednisone in another. One patient showed no signs of GVHD; the onset of GVHD took place between days +8 and +18, and was grade II-III in the other four. Three patients had limited chronic GVHD. Four patients are alive and in good clinical conditions at 11 to 63 months post-BMT. The remainder, who had stage III GVHD, died on day +45. The favourable results of BMT in FA have been confirmed, so this procedure should be considered as the best choice in the treatment of FA.