Purpose Allogeneic hematopoietic cell transplantation (HCT) can cure bone marrow failure in patients with Fanconi anemia (FA). Data on outcomes in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome (MDS), or acute leukemia have not been separately analyzed. Patients and Methods We analyzed data on 113 patients with FA with cytogenetic abnormalities (n 54), MDS (n 45), or acute leukemia (n 14) who were reported to the Center for International Blood and Marrow Transplant Research from 1985 to 2007. Results Neutrophil recovery occurred in 78% and 85% of patients at days 28 and 100, respectively. Day 100 cumulative incidences of acute graft-versus-host disease grades B to D and C to D were 26% (95% CI, 19% to 35%) and 12% (95% CI, 7% to 19%), respectively. Survival probabilities at 1, 3, and 5 years were 64% (95% CI, 55% to 73%), 58% (95% CI, 48% to 67%), and 55% (95% CI, 45% to 64%), respectively. In univariate analysis, younger age was associated with superior 5-year survival ( v 14 years: 69% [95% CI, 57% to 80%] v 39% [95% CI, 26% to 53%], respectively; P .001). In transplantations from HLA-matched related donors (n 82), younger patients ( v 14 years: 78% [95% CI, 64% to 90%] v 34% [95% CI, 20% to 50%], respectively; P .001) and patients with cytogenetic abnormalities only versus MDS/acute leukemia (67% [95% CI, 52% to 81%] v 43% [95% CI, 27% to 59%], respectively; P .03) had superior 5-year survival. Conclusion Our analysis indicates that long-term survival for patients with FA with cytogenetic abnormalities, MDS, or acute leukemia is achievable. Younger patients and recipients of HLA-matched related donor transplantations who have cytogenetic abnormalities only have the best survival.
Bibliographical noteFunding Information:
The Center for International Blood and Marrow Transplant Research is supported by Public Health Service Grant/Cooperative Agreement No. U24-CA76518 from the National Cancer Institute (NCI), the National Heart, Lung, and Blood Institute (NHLBI), and the National Institute of Allergy and Infectious Diseases; Grant/Cooperative Agreement No. 5U01HL069294 from NHLBI and NCI; Contract No. HHSH234200637015C from the Health Resources and Services Administration and Department of Health and Human Services; Grants No. N00014-06-1-0704 and N00014-08-1-0058 from the Office of Naval Research; and grants from Allos; Amgen; Angioblast; anonymous donation to the Medical College of Wisconsin; Ariad; Be the Match Foundation; Blue Cross and Blue Shield Association; Buchanan Family Foundation; CaridianBCT; Celgene Corporation; CellGenix; Children’s Leukemia Research Association; Fresenius-Biotech North America; Gamida Cell Teva Joint Venture; Genentech; Genzyme; GlaxoSmithKline; Histo-Genetics; Kiadis Pharma; The Leukemia and Lymphoma Society; The Medical College of Wisconsin; Merck; Millennium: The Takeda Oncology Company; Milliman USA; Miltenyi Biotec; National Marrow Donor Program; Optum Healthcare Solutions; Osiris Therapeutics; Otsuka America Pharmaceutical; RemedyMD; Sanofi; Seattle Genetics; Sigma-Tau Pharmaceuticals; Soligenix; StemCyte; Stemsoft Software; Swedish Orphan Biovit-rum; Tarix Pharmaceuticals; Teva Neuroscience; THERAKOS; and Wellpoint.