Treatment of the bone marrow failure in Fanconi anemia patients with danazol
K Scheckenbach, M Morgan, J Filger-Brillinger… - Blood Cells, Molecules …, 2012 - Elsevier
More than 90% of Fanconi anemia (FA) patients experience progressive bone marrow
failure during life with a median onset at 8years of age. As matched sibling donor
transplantation as preferred treatment is not available for the majority of patients, several
synthetic androgens have been used as short-term treatment options for the marrow failure
in FA patients for more than 50years. Here, we retrospectively collected data on eight FA
patients who received danazol for the off-label treatment of their marrow failure at a starting …
failure during life with a median onset at 8years of age. As matched sibling donor
transplantation as preferred treatment is not available for the majority of patients, several
synthetic androgens have been used as short-term treatment options for the marrow failure
in FA patients for more than 50years. Here, we retrospectively collected data on eight FA
patients who received danazol for the off-label treatment of their marrow failure at a starting …
