Allogeneic hematopoietic stem cell transplantation in Fanconi anemia: The European Group for Blood and Marrow Transplantation experience

Régis Peffault De Latour, Raphael Porcher, Jean Hugues Dalle, Mahmoud Aljurf, Elisabeth T. Korthof, Johanna Svahn, Roelof Willemze, Cristina Barrenetxea, Valerie Mialou, Jean Soulier, Mouhab Ayas, Rosi Oneto, Andrea Bacigalupo, Judith C W Marsh, Christina Peters, Gerard Socie, Carlo Dufour

Research output: Contribution to journalArticlepeer-review

Abstract

Although allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment for patients with Fanconi anemia (FA), published series mostly refer to single-center experience with limited numbers of patients. We analyzed results in 795 patients with FA who underwent first HSCT between May 1972 and January 2010. With a 6-year median follow-up, overall survival was 49% at 20 years (95% confidence interval, 38-65 years). Better outcome was observed for patients transplanted before the age of 10 years, before clonal evolution (ie, myelodysplastic syndrome or acute myeloid leukemia), from a matched family donor, after a conditioning regimen without irradiation, the latter including fludarabine. Chronic graft-versus-host disease and secondary malignancy were deleterious when considered as time-dependent covariates. Age more than 10 years at time of HSCT, clonal evolution as an indication for transplantation, peripheral blood as source of stem cells, and chronic graft-versus-host disease were found to be independently associated with the risk for secondary malignancy. Changes in transplant protocols have significantly improved the outcome of patients with FA, who should be transplanted at a young age, with bone marrow as the source of stem cells.

Original languageEnglish
Pages (from-to)4279-4286
Number of pages8
JournalBlood
Volume122
Issue number26
DOIs
Publication statusPublished - Dec 19 2013

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

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