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

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

Transplantation and Cellular Therapy, June 2015

26116087

Mouhab 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

Second allogeneic hematopoietic cell transplantation (HCT) is the only salvage option for those for develop graft failure after their first HCT. Data on outcomes after second HCT in Fanconi anemia (FA) are scarce. We report outcomes after second allogeneic HCT for FA (n=81). The indication for second HCT was graft failure after the first HCT. Transplants occurred between 1990 and 2012. The timing of second transplantation predicted subsequent graft failure and survival. Graft failure was high when the second transplant occurred less than 3 months from the first. The 3-month probability of graft failure was 69% when the interval between first and second transplant was less than 3 months compared to 23% when the interval was longer (p<0.001). Consequently, survival rates were substantially lower when the interval between first and second transplant was less than 3 months, 23% at 1-year compared to 58%, when the interval was longer (p=0.001). The corresponding 5-year probabilities of survival were 16% and 45%, respectively (p=0.006). Taken together, these data suggest that fewer than half of FA patients undergoing a second HCT for graft failure are long-term survivors. There is an urgent need to develop strategies to lower graft failure after first HCT.