We retrospectively analyzed results of unrelated cord blood transplantation (UCBT) in 93 Fanconi anemia (FA) patients. Median age at transplantation was 8.6 years (1-45). The units transplanted were HLA-A, -B, or -DRB1 identical in 12 cases, 1 HLA mismatch in 35 cases, and 2 or 3 HLA differences in 45 cases. The median number of nucleated cells (NC) and CD34+ cells infused of recipient weight was 4.9 × 107/kg and 1.9 × 105/kg, respectively. Participating centers selected the preparative regimen of their choice, in 57 patients (61%), it included Fludarabine. Graft-versus-host disease (GVHD) prophylaxis consisted mostly of cyclosporine with prednisone. Cumulative incidence (CI) of neutrophil recovery was 60 ± 5% at day +60. In multivariate analysis, Fludarabine containing regimen and NC infused ≥4.9 × 107/kg were associated with higher probability of recovery. CI of grade II-IV acute and of chronic GVHD (aGVHD, cGVHD) was 32% ± 5% and 16% ± 4%, respectively. Overall survival (OS) was 40% ± 5%. In multivariate analysis, factors associated with favorable outcome were use of Fludarabine in the conditioning regimen, number of NC infused ≥4.9 × 107/kg, and negative cytomegalovirus (CMV) serology in the recipient. In conclusion, factors easily modifiable such as donor selection and a Fludarabine-containing regimen can considerably improve survival in FA patients given a UCBT. These data are the basis for designing prospective protocols.
Bibliographical noteFunding Information:
Eurocord is supported by a European Grant QLK3-CT-1999-00380. We are grateful to the Eurocord transplant centers (listed in the appendix ), and to all the data managers and research coordinators for their invaluable help with the data collection. We also thank all the Netcord and other cord blood banks (listed in the appendix ). We would like to thank again all the coauthors for their contributions and critical reading of this manuscript: Eliane Gluckman designed the study and wrote the paper. Vanderson Rocha collected the data, performed the statistical analysis, and contributed to the writing of the article. Irina Ionescu collected and validated the data, entered them in our data base, and contributed to the writing of the article. M. Bierings, R.E. Harris, J. Wagner, J. Kurtzberg, M.A. Champagne, C. Bonfim, M. Bittencourt, P. Darbyshire, M.N. Fernandez, F. Locatelli, and R. Pasquini transplanted the patients and sent the clinical data. They all contributed and edited this manuscript. The authors declare no competing financial interests.
- Fanconi anemia
- Unrelated cord blood transplantation