Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia

Mouhab Ayas, Wael Saber, Stella M. Davies, Richard E. Harris, Gregory A. Hale, Gerard Socie, Jennifer Lerademacher, Monica Thakar, H. Joachim J. Deeg, Amal Al-Seraihy, Minoo Battiwalla, Bruce M. Camitta, Richard Olsson, Rajinder S. Bajwa, Carmem M. Bonfim, Ricardo Pasquini, Margaret L. MacMillan, Biju George, Edward A. Copelan, Baldeep WirkAbdullah Al Jefri, Anders L. Fasth, Eva C. Guinan, Biljana N. Horn, Victor A. Lewis, Shimon Slavin, Polina Stepensky, Marc Bierings, Robert Peter Gale

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41 Scopus citations

Abstract

Purpose Allogeneic hematopoietic cell transplantation (HCT) can cure bone marrow failure in patients with Fanconi anemia (FA). Data on outcomes in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome (MDS), or acute leukemia have not been separately analyzed. Patients and Methods We analyzed data on 113 patients with FA with cytogenetic abnormalities (n 54), MDS (n 45), or acute leukemia (n 14) who were reported to the Center for International Blood and Marrow Transplant Research from 1985 to 2007. Results Neutrophil recovery occurred in 78% and 85% of patients at days 28 and 100, respectively. Day 100 cumulative incidences of acute graft-versus-host disease grades B to D and C to D were 26% (95% CI, 19% to 35%) and 12% (95% CI, 7% to 19%), respectively. Survival probabilities at 1, 3, and 5 years were 64% (95% CI, 55% to 73%), 58% (95% CI, 48% to 67%), and 55% (95% CI, 45% to 64%), respectively. In univariate analysis, younger age was associated with superior 5-year survival ( v 14 years: 69% [95% CI, 57% to 80%] v 39% [95% CI, 26% to 53%], respectively; P .001). In transplantations from HLA-matched related donors (n 82), younger patients ( v 14 years: 78% [95% CI, 64% to 90%] v 34% [95% CI, 20% to 50%], respectively; P .001) and patients with cytogenetic abnormalities only versus MDS/acute leukemia (67% [95% CI, 52% to 81%] v 43% [95% CI, 27% to 59%], respectively; P .03) had superior 5-year survival. Conclusion Our analysis indicates that long-term survival for patients with FA with cytogenetic abnormalities, MDS, or acute leukemia is achievable. Younger patients and recipients of HLA-matched related donor transplantations who have cytogenetic abnormalities only have the best survival.

Original languageEnglish (US)
Pages (from-to)1669-1676
Number of pages8
JournalJournal of Clinical Oncology
Volume31
Issue number13
DOIs
StatePublished - May 1 2013

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ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Ayas, M., Saber, W., Davies, S. M., Harris, R. E., Hale, G. A., Socie, G., Lerademacher, J., Thakar, M., Deeg, H. J. J., Al-Seraihy, A., Battiwalla, M., Camitta, B. M., Olsson, R., Bajwa, R. S., Bonfim, C. M., Pasquini, R., MacMillan, M. L., George, B., Copelan, E. A., ... Gale, R. P. (2013). Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia. Journal of Clinical Oncology, 31(13), 1669-1676. https://doi.org/10.1200/JCO.2012.45.9719