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Second Allogeneic Hematopoietic Cell Transplantation for Patients with Fanconi Anemia and Bone Marrow Failure.

Abstract
A second allogeneic hematopoietic cell transplantation (HCT) is the sole salvage option for individuals who develop graft failure after their first HCT. Data on outcomes after second HCT in patients with Fanconi anemia (FA) are scarce. Here we report outcomes after second allogeneic HCT for FA (n = 81). The indication for second HCT was graft failure after the first HCT. Transplantations were performed between 1990 and 2012. The timing of the second HCT predicted subsequent graft failure and survival. Graft failure was high when the second HCT was performed less than 3 months from the first. The 3-month probability of graft failure was 69% when the interval between the first HCT and second HCT was less than 3 months, compared with 23% when the interval was longer (P < .001). Consequently, the 1-year survival rate was substantially lower when the interval between the first and second HCTs was less than 3 months compared with longer (23% vs 58%; P = .001). The corresponding 5-year probability of survival was 16% and 45%, respectively (P = .006). Taken together, these data suggest that fewer than one-half of patients with FA undergoing a second HCT for graft failure are long-term survivors. There is an urgent need to develop strategies to reduce the rate of graft failure after first HCT.
AuthorsMouhab Ayas, Mary Eapen, Jennifer Le-Rademacher, Jeanette Carreras, Hisham Abdel-Azim, Blanche P Alter, Paolo Anderlini, Minoo Battiwalla, Marc Bierings, David K Buchbinder, Carmem Bonfim, Bruce M Camitta, Anders L Fasth, Robert Peter Gale, Michelle A Lee, Troy C Lund, Kasiani C Myers, Richard F Olsson, Kristin M Page, Tim D Prestidge, Mohamed Radhi, Ami J Shah, Kirk R Schultz, Baldeep Wirk, John E Wagner, H Joachim Deeg
JournalBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (Biol Blood Marrow Transplant) Vol. 21 Issue 10 Pg. 1790-5 (Oct 2015) ISSN: 1523-6536 [Electronic] United States
PMID26116087 (Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't, Research Support, U.S. Gov't, Non-P.H.S.)
CopyrightCopyright © 2015 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
Topics
  • Adolescent
  • Allografts
  • Child
  • Child, Preschool
  • Fanconi Anemia (therapy)
  • Female
  • Graft Rejection (mortality, therapy)
  • Graft vs Host Disease (etiology, mortality)
  • Hematopoietic Stem Cell Transplantation (statistics & numerical data)
  • Humans
  • Infant
  • Infections (mortality)
  • Kaplan-Meier Estimate
  • Leukocyte Count
  • Lymphoproliferative Disorders (etiology)
  • Male
  • Multiple Organ Failure (etiology, mortality)
  • Neutrophils
  • Salvage Therapy
  • Survival Rate
  • Young Adult

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