Five-year 5-year overall survival was 87% in a single-center study of 103 consecutive patients with Fanconi anemia who underwent allogeneic bone marrow transplantation between 2003 and 2014 using chemotherapy-only conditioning. Patients were a median age of 9 years (range, 3-23), and transplant grafts were matched sibling donors (MSD, n=49), other related donors (ORD, n=12), 8/8 matched unrelated donors (URD, n=36), and 7/8 matched unrelated donors (MURD, n=6).
There was no difference in survival according to the type of donor: 92%, 92%, and 83% for MSD, ORD, and unrelated donors (URD + MURD), respectively. Patients without acute GVHD had an overall survival of 98% compared to 58% in patients with acute GVHD (p=0.001).
Lead researcher Dr. Carmem Bonfim of the Federal University of Paraná, Curitiba, Brazil, concluded that “the use of non-irradiation protocols was associated with an excellent survival, successful engraftment, and very low mortality rate.”