BONE-MARROW TRANSPLANTATION IN FANCONI-ANEMIA USING MATCHED SIBLING DONORS

Eighteen patients with Fanconi anemia (FA) with evidence of bone marro w (BM) aplasia underwent allogenic BM transplants (BMT) from matched s ibling donors (MSD). Median age at BMT was 7.6 years, Conditioning con sisted of low-dose cyclophosphamide (CY; 5 mg/kg x 4 days) and thoraco abdominal irradiation (TAI; 400 cGy). Graft-versus-host disease (GVHD) prophylaxis included cyclosporin A and prednisone. In addition antith ymocyte globulin (ATG) was administered in the pretransplant period to promote engraftment and in the posttransplant period for additional G VHD prophylaxis. Engraftment occurred rapidly (median, 12 days for an absolute neutrophil count greater than or equal to 0.5 x 10(9)/L; medi an, 22 days for platelet count greater than or equal to 50 x 10(9)/L). Seventeen patients have sustained engraftment and are transfusion-ind ependent, with Lansky scores of 100% at median follow-up of 27 months. One patient developed graft failure 4 months after initial engraftmen t and required a second BM infusion. None of the patients developed ac ute GVHD; 3 patients (16%) developed chronic GVHD. BMT is a feasible o ption for FA patients having an MSD and should be performed at a young age and early in the course of the disease, before the development of complications. We believe the addition of ATG to the transplant regim en of low-dose CY, TAI, and cyclosporin was responsible for improvemen t in the survival of FA patients undergoing BMT. The regimen was well tolerated and was associated with a low incidence of complications inc luding GVHD. (C) 1994 by The American Society of Hematology.