Abstract
Cessation of androgen therapy and successful allogeneic bone marrow transplantation in a boy with IAAA was followed by regression of his oxymetholone-induced hepatic tumors. This 13-year-old boy developed severe IAAA in 1972. He experienced a remission following therapy with oxymetholone and prednisone. After 34 months of oxymetholone he developed jaundice, an enlarged tender liver and elevated liver enzymes. Liver scans showed 3 intrahepatic filling defects. Because of thrombocytopenia, biopsy was not performed. A presumptive diagnosis of androgen-associated hepatoma was made and oxymetholone was discontinued. The patient's IAAA promptly relapsed and allogeneic bone marrow transplantation from his HLA-A, B, and D identical sister was performed following preparation with cyclophosphamide (200 mg/kg). Liver scans performed before and immediately after cyclophosphamide showed no change. The post-transplant course was benign and full hematologic reconstitution with donor cells occurred within 4 weeks. There were no signs of a graft-versus-host reaction. Follow-up liver scans have shown gradual disappearance of the filling defects and liver enzymes have dropped. The patient continues to do wcl1 6 months after transplantation. This experience further supports the argument for early transplantation rather than androgen therapy whenever possible for patients with IAAA.
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Montgomery, R., Ducore, J., Githens, J. et al. REGRESSION OF OXYMETHOLONE INDUCED HEPATIC TUMORS FOLLOWING ALLOGENEIC BONE MARROW TRANSPLANTATION FOR IDIOPATHIC ACQUIRED APLASTIC ANEMIA (IAAA). Pediatr Res 11, 477 (1977). https://doi.org/10.1203/00006450-197704000-00646
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DOI: https://doi.org/10.1203/00006450-197704000-00646