PEDIATRIC BLOOD & CANCER, vol.63, no.4, pp.695-700, 2016 (SCI-Expanded)
BackgroundFanconi anemia (FA) is a heterogeneous autosomal recessive (and rarely X linked) disorder, which is characterized by congenital malformations, progressive bone marrow failure, and predisposition to malignancies. Hematopoietic stem cell transplantation (HSCT) is the only definitive treatment for the hematological manifestations in FA.