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.
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Financial disclosure: This research was supported in part by the Intramural Research Program of the National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI). The Center for International Blood and Marrow Transplant Research is supported by Public Health Service Grant/Cooperative Agreement U24-CA076518 from the National Cancer Institute (NCI), NHLBI , and National Institute of Allergy and Infectious Diseases ; Grant/Cooperative Agreement 5U10HL069294 from the NHLBI and NCI ; Contract HHSH250201200016C with the Health Resources and Services Administration; Grants N00014-13-1-0039 and N00014-14-1-0028 from the Office of Naval Research ; and grants from Actinium Pharmaceuticals *; Allos Therapeutics, Inc ; Amgen *; Anonymous donation to the Medical College of Wisconsin ; Ariad ; Be The Match Foundation ; Blue Cross and Blue Shield Association *; Celgene *; Chimerix, Inc ; Fred Hutchinson Cancer Research Center ; Fresenius-Biotech North America, Inc ; Gamida Cell Teva Joint Venture Ltd *; Genentech ; Gentium SpA *; Genzyme ; GlaxoSmithKline ; Health Research, Inc, Roswell Park Cancer Institute ; HistoGenetics ; Incyte Corp ; Jeff Gordon Children's Foundation ; Kiadis Pharma ; Medac GmbH ; The Medical College of Wisconsin ; Merck & Co., Inc. ; Millennium: The Takeda Oncology Co ; Milliman USA, Inc *; Miltenyi Biotec *; National Marrow Donor Program; Onyx Pharmaceuticals ; Optum Healthcare Solutions, Inc ; Osiris Therapeutics ; Otsuka America Pharmaceutical, Inc ; PerkinElmer, Inc ; Remedy Informatics *; Sanofi US *; Seattle Genetics ; Sigma-Tau Pharmaceuticals ; Soligenix, Inc ; St. Baldrick's Foundation ; StemCyte, a Global Cord Blood Therapeutics Co ; Stemsoft Software, Inc ; Swedish Orphan Biovitrum ; Tarix Pharmaceuticals *; Terumo BCT *; Teva Neuroscience, Inc *; Therakos *; University of Minnesota ; University of Utah ; and WellPoint *. The views expressed in this article do not reflect the official policy or position of the NIH, the Department of the Navy, the Department of Defense, the Health Resources and Services Administration, or any other agency of the US Government. This research was supported in part by the Intramural Research Program of the NIH and the NCI (B.A.).
- Fanconi anemia
- Graft failure
- Second transplantation