Improved Outcomes in Alternative Donor HCT for Fanconi Anemia

This prospective study of 130 patients (age range 1-48) transplanted for Fanconi anemia (FA) has shown that over a period of 10 years, transplant toxicity has decreased and engraftment has improved, resulting in a 5-year overall survival (OS) of 58% (95% CI, 49-59). A 5-year OS of 94% was achieved in 17 patients without a history of opportunistic infection or transfusions, and whose conditioning regimens included fludarabine. The addition of fludarabine to conditioning regimens was associated with a threefold increase in engraftment and lower mortality risk at 5 years. All transplants used partially matched related (n=8) or unrelated (n=91) donors, or umbilical cord blood (n=31). The authors concluded that “most FA patients with standard risk disease are cured of their bone marrow failure by HCT even if an HLA-matched sibling donor is not available.”

MacMillan ML, et al. Blood

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