Treatment with deferiprone for iron overload alleviates bone marrow failure in a Fanconi anemia patient.

Fanconi anemia (FA) is a rare inherited disorder characterized by congenital abnormalities, progressive bone marrow failure and cancer susceptibility. There are no reports in the literature about a specific therapy effective in treating the progressive bone marrow failure of FA except for hematopoietic stem cell transplantation (HSCT). A FA patient started to receive deferiprone (L1) therapy due to iron overload. We report here that the white blood cell counts, hemoglobin (Hb) levels and platelet counts were significantly higher during the L1-treated period than when without L1 therapy. Therefore, L1 therapy may be worth considering for FA patients who cannot undergo HSCT.