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

作者：Ayas Mouhab^*; Eapen Mary; Le Rademacher Jennifer; Carreras Jeanette; Abdel Azim Hisham; Alter Blanche P; Anderlini Paolo; Battiwalla Minoo; Bierings Marc; Buchbinder David K; Bonfim Carmem; Camitta Bruce M; Fasth Anders L; Gale Robert Peter; Lee Michelle A; Lund Troy C; Myers Kasiani C; Olsson Richard F; Page Kristin M; Prestidge Tim D; Radhi Mohamed; Shah Ami J; Schultz Kirk R; Wirk Baldeep; Wagner John E; Deeg H Joachim

来源：Biology of Blood and Marrow Transplantation, 2015, 21(10): 1790-1795.

DOI：10.1016/j.bbmt.2015.06.012

• Fanconi anemia
• Graft failure
• Second transplantation

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.

• 出版日期2015-10
• 单位NIH