HLA-haploidentical T cell-depleted allogeneic hematopoietic stem cell transplantation in children with fanconi anemia

Marco Zecca, Luisa Strocchio, Daria Pagliara, Patrizia Comoli, Alice Bertaina, Giovanna Giorgiani, Cesare Perotti, Franco Corbella, Letizia Brescia, Franco Locatelli

Research output: Contribution to journalArticlepeer-review


We report the outcome of 12 consecutive pediatric patients with Fanconi anemia (FA) who had neither an HLA-identical sibling nor an HLA-matched unrelated donor and who were given Tcell-depleted, CD34+ positively selected cells from a haploidentical related donor after a reduced-intensity, fludarabine-based conditioning regimen. Engraftment was achieved in 9 of 12 patients (75%), and the cumulative incidence of graft rejection was 17% (95% confidence interval [CI], 5% to 59%). Cumulative incidences of grades II to IV acute and chronic graft-versus-host disease were 17% (95% CI, 5% to 59%) and 35% (95% CI, 14% to 89%), respectively. The conditioning regimen was well tolerated, with no fatal regimen-related toxicity and 3 cases of grade III regimen-related toxicity. The cumulative incidence of transplant-related mortality was 17% (95% CI, 5% to 59%). The 5-year overall survival, event-free survival, and disease-free survival were 83% (95% CI, 62% to 100%), 67% (95% CI, 40% to 93%), and 83% (95% CI, 62% to 100%), respectively. These data demonstrate that a fludarabine-based conditioning regimen, followed by infusion of high doses of Tcell-depleted stem cells, is able to ensure engraftment with good overall survival and disease-free survival, confirming the feasibility of haploidentical hematopoietic stem cell transplantation in FA. To the best of our knowledge, this is the largest series of hematopoietic stem cell transplantation from a haploidentical related donor in FA patients reported to date.

Original languageEnglish
Pages (from-to)571-576
Number of pages6
JournalBiology of Blood and Marrow Transplantation
Issue number4
Publication statusPublished - 2014


  • Fanconi anemia
  • Haploidentical stem cell transplantation
  • Reduced-intensity conditioning
  • Tcell depletion

ASJC Scopus subject areas

  • Hematology
  • Transplantation


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