Faculty Collaboration Database

Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia. Blood 2007 Mar 01;109(5):2256-62

Date

10/14/2006

Pubmed ID

17038525

Pubmed Central ID

PMC1801062

DOI

10.1182/blood-2006-07-036657

Scopus ID

2-s2.0-33847349880 (requires institutional sign-in at Scopus site)   163 Citations

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.

Author List

Wagner JE, Eapen M, MacMillan ML, Harris RE, Pasquini R, Boulad F, Zhang MJ, Auerbach AD

Authors

Mary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of Wisconsin
Mei-Jie Zhang PhD Professor in the Institute for Health and Equity department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Acute Disease
Adolescent
Adult
Blood Platelets
Bone Marrow Transplantation
Cell Proliferation
Child
Child, Preschool
Chronic Disease
Fanconi Anemia
Female
Graft vs Host Disease
Humans
Infant
Male
Neutrophils
Probability
Prognosis
Survival Rate
Tissue Donors