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Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia

Wagner, John E., Eapen, Mary, MacMillan, Margaret L., Harris, Richard E., Pasquini, Ricardo, Boulad, Farid, Zhang, Mei-Jie, Auerbach, Arleen D.·2007·Blood
DOI 10.1182/blood-2006-07-036657
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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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Metadata

Authors
Wagner, J. E., Eapen, M., MacMillan, M. L., Harris, R. E., Pasquini, R., Boulad, F., Zhang, M.-J., & Auerbach, A. D.
Affiliations
  • Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, University of Minnesota Blood and Marrow Transplant Program, Minneapolis;
  • Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee;
  • Cincinnati Children's Hospital, OH;
  • Hospital de Clinicas–Federal University of Parana, Brazil;
  • Memorial Sloan-Kettering Cancer Center, New York, NY;
  • Laboratory of Human Genetics and Hematology, Rockefeller University, New York, NY
Venue
Blood
Publisher
American Society of Hematology
Published
2006-10-12
Volume
109
Issue
5
Pages
2256-2262
ISSN
  • 0006-4971
  • 1528-0020
DOI
10.1182/blood-2006-07-036657
Cited by
165
References
0
Language
en

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CoChat. (2026). Citation check: Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia. https://app.cochat.ai/papers/10.1182/blood-2006-07-036657
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Wagner, J. E., Eapen, M., MacMillan, M. L., Harris, R. E., Pasquini, R., Boulad, F., Zhang, M.-J., & Auerbach, A. D. (2007). Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia. Blood. https://doi.org/10.1182/blood-2006-07-036657