The results of the oral administration of high doses of anabolic hormones (oxymetholone, metholone or dromostanolone and methenolone) in the treatment of 69 patients (14 children and 55 adults) with aplastic anemia are presented. Remission rates were 48 per cent for the whole group and 70 per cent for those treated for more than two months. Twenty-two cases have been in remission for 1 to 5 years without maintenance therapy. Eight others relapsed 2 to 4 months after stopping therapy, but responded to the reinstitution of medication. However, four of them have had several relapses and appear to require permanent therapy. In the other four, the second remission has continued for 6 to 40 months. Clinical response was first noted after 0.6 to 6 months of therapy and was characterized by a rise of hemoglobin to normal levels with a variable degree of improvement in the leukopenia and thrombocytopenia. The number of neutrophils and platelets rose to normal levels in over a third of the cases which responded. Response to therapy was not related to age, sex, etiology, degree of pancytopenia or bone marrow cellularity. The three anabolic hormones seemed equally effective.
Nine patients became clinically jaundiced during therapy; four recovered and five died. Abnormalities in liver function existed before treatment in three of those who died. In four of these five cases, the liver lesion was due to an independent cause: viral hepatitis in one, gram negative septicemia and necrotic colitis in two and thiazide toxicity in the fourth. No autopsy was performed in the other case and the etiology of his liver damage could not be determined. Three of the fatal cases were taking a non-17-alkylated steroid. Other patients presented mild, subclinical and nonprogressive rises in serum direct bilirubin and in BSP retention. The frequency of liver function changes was greater (80 per cent) with oxymetholone than with the other two non-17-alkylated steroids (26 per cent). The changes induced with the former drug regressed completely when metholone or methenolone was substituted. Other side effects were amenorrhea and mild virilization; no growth retardation or changes in bone maturation were observed.
Our results suggest that some anabolic adrogenic steroids may be more useful than testosterone in the treatment of aplastic anemia.