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Bone marrow transplantation in Fanconi anemia using matched sibling donors
M Kohli-Kumar, C Morris, C DeLaat, J Sambrano, M Masterson, R Mueller, NT Shahidi, G Yanik, K Desantes and DJ Friedman
Bone Marrow Transplant Program, Children's Hospital Medical Center,
Cincinnati, OH 45229.
Eighteen patients with Fanconi anemia (FA) with evidence of bone marrow
(BM) aplasia underwent allogenic BM transplants (BMT) from matched sibling
donors (MSD). Median age at BMT was 7.6 years. Conditioning consisted of
low-dose cyclophosphamide (CY; 5 mg/kg x 4 days) and thoracoabdominal
irradiation (TAI; 400 cGy). Graft-versus-host disease (GVHD) prophylaxis
included cyclosporin A and prednisone. In addition antithymocyte globulin
(ATG) was administered in the pretransplant period to promote engraftment
and in the posttransplant period for additional GVHD prophylaxis.
Engraftment occurred rapidly (median, 12 days for an absolute neutrophil
count > or = 0.5 x 10(9)/L; median, 22 days for platelet count > or =
50 x 10(9)/L). Seventeen patients have sustained engraftment and are
transfusion-independent, with Lansky scores of 100% at median follow-up of
27 months. One patient developed graft failure 4 months after initial
engraftment and required a second BM infusion. None of the patients
developed acute GVHD; 3 patients (16%) developed chronic GVHD. BMT is a
feasible option 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 regimen of
low-dose CY, TAI, and cyclosporin was responsible for improvement in the
survival of FA patients undergoing BMT. The regimen was well tolerated and
was associated with a low incidence of complications including GVHD.
Volume 84,
Issue 6,
pp. 2050-2054,
09/15/1994
Copyright © 1994 by The American Society of Hematology
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