OXYMETHOLONE
Potent oral 17α-alkylated anabolic steroid used for anemia and wasting; high hepatotoxicity risk.
Oxymetholone was FDA-approved in the 1960s for treating anemias caused by deficient red cell production, and has been studied off-label for HIV-associated wasting, antithrombin III deficiency, and pediatric growth impairment with varying success. Clinical evidence supports its erythropoietic effects, but pharmacokinetic data from pre-approval studies are limited. Hepatotoxicity is the most significant adverse effect, with cholestatic jaundice, peliosis hepatis, and hepatic tumor formation documented. The NTP lists oxymetholone as reasonably anticipated to be a human carcinogen. Animal studies confirm testicular toxicity and impaired spermatogenesis.