Understanding Factors in Long-Term HCT Outcome in Fanconi Anemia
The estimated 20-year survival of 795 Fanconi anemia (FA) patients who underwent first HCT between 1972 and 2010 is 49%, according to this multi-center study published in Blood. Significantly better outcomes are achieved in FA patients under age 10 years and before clonal evolution to myelodysplastic syndromes or AML. Other factors leading to better survival are HCT from a matched family donor, and conditioning regimens without irradiation and with fludarabine. Age more than 10 years at time of HCT, clonal evolution as an indication for transplant, peripheral blood grafts, and chronic GVHD were independently associated with the risk of late secondary malignancies. The authors conclude that bone marrow transplant at a young age yields the best outcomes in FA patients.