Anabolic Androgenic Steroids in the Treatment of Acquired Aplastic Anemia

Abstract

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.

Treatment of Refractory Anemias with Methenolone

“…When using androgenic-anabolic steroids the reported remission frequencies lie around 50% (7,9,11,12). In the small series reported here, the results are of a similar magnitude.…”

“…Since the introduction of androgenic-anabolic steroids in the therapy of aplastic anemias by Coletta et al (2) and Seip (13) in 1961, the majority of reports concern oxymetholone ( I 1, 12). The effects of this drug in different anemias are quite well documented; its most serious drawback is its liver toxicity ( I I , 12). Recently liver tumours have been reported after treatment with oxymetholone ( I ) .…”

“…The androgenic-anabolic steroids nandrolone and methenolone seem to have similar antianemic properties but a lower liver toxicity than oxymetholone (11,12). Reports on the use of these drugs are scanty, especially with respect to long-term effects.…”

Oxymetholone in the Treatment of Anaemia in Chronic Renal Failure

“…At the dose of 100 mg. oxymetholone daily, which is much higher than that usually employed for anabolic action, we did not encounter liver toxicity. However, we would be hesitant to repeat the trial at a yet higher dosage since the use of oxymetholone in doses of several hundred mg. per day for hypoplastic anaemia has been attended by jaundice and deranged liver function tests in up to 80 per cent of patients (Sanchez-Medal et a/., 1969). Liver function usually returns to normal within a few weeks of withdrawing the drug but the administrative upheaval caused by any case of jaundice in a hepatitis-conscious dialysis unit is too great to justify the risk.…”

“…We demonstrated no benefit from oxymetholone in this safe dosage for 3 months. Response to oxymetholone and other androgenic drugs in hypoplastic anamia is often delayed by a period of 2 weeks to 6 months (Sanchez-Medal et al, 1969). However the great majority of those who are going to respond have started to do so within 3 months and the same applies to patients in renal failure receiving testosterone (DeGowin et al, 1970;Richardson and Weinstein, 1970;Shaldon et al, 1970Shaldon et al, , 1971a.…”