Long-Term Follow-up of Outcomes of Hematopoietic Stem Cell Transplantation for Patients with Fanconi Anemia: A Single Center Retrospective Review

Fanconi anemia (FA) is an inherited bone marrow failure syndrome caused by defects in DNA repair. Patients often require hematopoietic stem cell transplantation (HSCT) to cure their severe bone marrow failure. Owing to the defects in DNA repair, patients with Fanconi anemia have significant morbidity with myeloablative conditioning prior to HSCT. Previous studies have reported good outcomes with reduced intensity conditioning (RIC) prior to HSCT for patients with FA. There are limited studies that compare outcomes between matched related donors and alternative donors.