Regression of androgen-related hepatic tumors in patients with Fanconi's anemia following marrow transplantation.

Two patients with Fanconi's anemia treated for 5 years with oxymetholone developed hepatic function abnormalities in association with hepatic tumors demonstrated by isotope liver-spleen scan or abdominal echogram. The lesions resolved over a period of 26 months after allogeneic marrow transplantation, and the patients are alive and well 3 and 4 years following transplantation. The course of these patients indicates that marrow transplantation for Fanconi's anemia allows the withdrawal of androgens and subsequent regression of androgen-related hepatic tumors in patients who might otherwise have a fatal outcome.