Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia

The Rockefeller University, New York, New York, United States
Blood (Impact Factor: 9.78). 04/2007; 109(5):2256-62. DOI: 10.1182/blood-2006-07-036657
Source: PubMed

ABSTRACT Bone marrow transplantation (BMT) is the only known cure for the hematologic manifestations of Fanconi anemia (FA). Potential benefits of unrelated donor BMT for FA, however, have been severely limited by graft rejection and treatment-related mortality with resultant poor survival. Therefore, we evaluated the impact of potential prognostic factors on hematopoietic recovery, graft-versus-host disease (GVHD), and mortality in 98 recipients of unrelated donor BMT who received transplants between 1990 and 2003. Probabilities of neutrophil (89% vs 69%; P = .02) and platelet (74% vs 23%; P < .001) recovery were higher after fludarabine-containing regimens than nonfludarabine-containing regimens. Risks of acute GVHD (relative risk [RR], 4.29; P < .001) were higher with non-T-cell-depleted grafts. The day-100 mortality rate was significantly higher after nonfludarabine-containing regimens than fludarabine-containing regimens (65% vs 24%, respectively; P < .001). Corresponding 3-year adjusted overall survival rates were 13% versus 52% (P < .001). In addition, mortality was higher in recipients who were older (> 10 years), who were cytomegalovirus (CMV) seropositive, and who received more than 20 blood product transfusions before BMT. Based on these results, significant practice changes are suggested: use of a fludarabine-containing conditioning regimen in the context of T-cell-depleted marrow allografts, and earlier referral for transplantation prior to excessive transfusions in patients with marrow failure.

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    ABSTRACT: The guidelines presented in this article have been prepared and approved in the I Meeting of Brazilian Guidelines in Hematopoietic Stem Cell Transplantation (HSCT) - Rio de Janeiro, July 19-21, 2009. The event was sponsored by SBTMO (Brazilian Society of Bone Marrow Transplantation). In this paper, we treat the severe aplastic anemia (SAA), considered a hematological emergency, that when identified and medically treated early, shows a great chance of recovery of the hematopoiesis, either through bone marrow transplantation or immunosuppressive therapy. Its objective is to guide the management of the transplantation, and indicate methods of conditioning, according to clinical characteristics of each patient, including the number of transfusions, in order to minimize the primary and secondary rejection, ensuring better overall and disease-free survival observed in literature and already validated by the results in our population. In the Fanconi Anemia, transplantation is the only curative option for the aplastic bone marrow component, although insignificant for the other components of the syndrome, it also requires skill and agility in finding a donor with important results.
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