In this multicenter, retrospective study of adults with Fanconi anemia (FA) undergoing hematopoietic cell transplantation (HCT) between 1991 and 2014, researchers found that best outcomes were in patients who received bone marrow from an identical sibling with fludarabine in the conditioning regimen and transplanted after the year 2000.
The 199 patients in the study had a median age of 16 years when diagnosed with FA, and underwent transplantation at a median age of 23 years. At time of transplant, 54% of patients had bone marrow failure and 46% had clonal disease.
Median follow-up was 58 months. Engraftment occurred in 82% of recipients, grade II-IV acute graft-versus-host disease (GVHD) occurred in 22%, and incidence of chronic GVHD at 96 months was 26%.
Patients achieved an overall survival (OS) rate of 84% at 36 months for those who received marrow from an identical sibling or matched unrelated donor. This is a significant improvement from an OS of 34% for patients who underwent HCT prior to year 2000.