C tier · mixed
It is the only compound here that retains a live FDA-approved indication (anaemia from deficient red cell production), which anchors a real regulatory evidence file, but the supporting clinical literature is largely pre-modern, small, and predates contemporary trial standards.
oxymetholone
Oxymetholone retains an approved haematological indication and is named explicitly in NIH LiverTox as one of the agents associated with roughly 1% incidence of acute cholestasis. Its efficacy record in anaemia rests substantially on older literature rather than large contemporary randomized trials, and it is widely regarded as among the most hepatotoxic agents in this class.
the explanation
Oxymetholone is a strong oral steroid still approved in the US to treat certain anaemias, meaning it has a legitimate medical role. It is also one of the harshest of these drugs on the liver, and health agencies list it by name among the steroids that cause bile-flow blockage and jaundice.
regulatory status
US Schedule III controlled substance; UK Class C (Misuse of Drugs Act). Retains an approved US medical indication.
Oxymetholone is a Schedule III controlled substance under the US Controlled Substances Act and a Class C drug under the UK Misuse of Drugs Act. Unlike oxandrolone — whose US approval was withdrawn in 2023 and formally determined to have been withdrawn for reasons of safety or effectiveness — oxymetholone historically held and, per reference sources, retains FDA approval, with the remaining indication being the treatment of anaemias caused by deficient red cell production. Current active marketing status in the US could not be confirmed from the primary label database during this review and is flagged as unverified.
how it works · proposed mechanism
Oxymetholone is an androgen receptor agonist whose principal approved therapeutic effect is stimulation of erythropoiesis.
Stimulation of erythropoiesis
Androgens increase erythropoietin production and enhance the response of erythroid progenitors in bone marrow, raising haemoglobin and haematocrit. This is the pharmacological basis for the compound's surviving indication in anaemia of deficient red cell production.
Androgen receptor agonism
As a 5α-reduced DHT derivative, oxymetholone binds the androgen receptor without requiring 5α-reductase activation and cannot serve as an aromatase substrate at the A-ring in the conventional manner. Nitrogen retention and increased protein synthesis follow receptor-mediated transcriptional changes.
17α-alkylation and liver load
The 17α-methyl group confers oral bioavailability by resisting hepatic first-pass oxidation, concentrating drug exposure on hepatocytes. LiverTox names oxymetholone specifically among agents in which acute cholestasis occurs in approximately 1% of treated patients.
what’s reported
evidence shape
5 sourcesThe composition of the cited literature by study type. This describes the shape of the evidence, not its quality — and it does not by itself set the grade.
⚠ the catch
Retaining an approved indication is a regulatory fact, not a statement that the modern efficacy evidence is strong — the haematological literature supporting it is largely old, small, and has been superseded in practice by erythropoiesis-stimulating agents and immunosuppression. Meanwhile it is explicitly named by NIH LiverTox in the ~1% cholestasis figure, so the harm evidence is in some respects more specific than the benefit evidence.
key published findings
- Human (NIH LiverTox): acute cholestasis is dose-related and occurs in approximately 1% of patients treated with methyltestosterone, danazol, stanozolol or oxymetholone — oxymetholone is named explicitly among the four.
- Human (NIH LiverTox): cholestasis has NOT been described in patients receiving unmodified testosterone, isolating 17α-alkylation as the structural driver of this injury.
- Human (NIH LiverTox): peliosis hepatis develops after 2-27 months of androgen therapy and may present asymptomatically or with acute hepatic rupture and haemorrhage.
- Human (NIH LiverTox): hepatic adenoma and hepatocellular carcinoma are associated with long-term androgen use — historically during therapy of aplastic anaemia or hypogonadism, precisely oxymetholone's indication population — typically after 5-15 years, with some cases within 2 years.
- Human observational (Baggish, Circulation 2017, n=140): long-term AAS users showed LVEF 52±11% versus 63±8% in non-users (p<0.001), a class-level cardiac signal relevant to any long-term oral androgen exposure.
limitations of the evidence
- No large modern randomized controlled trial of oxymetholone was verifiable within this review; specific trial-level efficacy numbers are therefore not reported rather than estimated.
- Its published elimination half-life is not reliably established in accessible sources and is recorded as null.
- Much of the class-level safety data pools several 17α-alkylated agents, so compound-specific attribution of risk magnitude is imprecise.
documented safety signals
- HEPATOTOXICITY — NAMED AGENT: NIH LiverTox names oxymetholone explicitly among agents in which dose-related acute cholestatic jaundice occurs in approximately 1% of treated patients, with onset typically 1-4 months and delayed presentations up to 6-24 months.
- PELIOSIS HEPATIS: blood-filled hepatic sinusoidal cysts documented at 2-27 months of therapy, capable of asymptomatic progression to fatal hepatic rupture and intra-abdominal haemorrhage. Historically a boxed-warning-level concern for this compound.
- HEPATIC ADENOMA AND HEPATOCELLULAR CARCINOMA: LiverTox associates long-term androgen therapy with hepatic tumours arising most often in the aplastic-anaemia and hypogonadism populations — the same populations treated with oxymetholone — typically after 5-15 years.
- SERUM ENZYME ELEVATION: LiverTox reports mean ALT ~57 U/L in actively using bodybuilders versus ~19 U/L in never-users, and bromsulfophthalein retention in 62% of one prospective cohort without symptoms, indicating subclinical hepatic dysfunction is common.
- ADVERSE LIPID EFFECTS: oral 17α-alkylated androgens markedly suppress HDL cholesterol and raise LDL via induction of hepatic triglyceride lipase, a class effect not shared by parenteral testosterone.
- CARDIOMYOPATHY AND ATHEROSCLEROSIS: Baggish et al. (Circulation 2017) documented reduced systolic function (LVEF 52±11% vs 63±8%, p<0.001), impaired diastolic relaxation (9.3±2.4 vs 11.1±2.0 cm/s, p<0.001), and dose-dependent coronary plaque burden in long-term AAS users.
- POLYCYTHAEMIA/ERYTHROCYTOSIS: the same erythropoietic mechanism that underpins the approved indication can raise haematocrit excessively, increasing blood viscosity and thrombotic risk.
- HPG AXIS SUPPRESSION: suppression of GnRH and pituitary LH/FSH with consequent testicular atrophy, impaired spermatogenesis and suppressed endogenous testosterone production.
- VIRILISATION IN WOMEN: hirsutism, voice deepening, male-pattern baldness, clitoral enlargement and menstrual disruption; voice change and clitoromegaly are frequently irreversible.
- GROWTH-PLATE EFFECTS IN ADOLESCENTS: acceleration of epiphyseal maturation with risk of premature growth-plate fusion and permanent reduction in attained adult height.
- FLUID RETENTION AND OESTROGENIC EFFECTS: oxymetholone is noted for pronounced fluid retention and gynaecomastia-type effects despite being non-aromatisable, attributed to intrinsic receptor cross-activity.
identity
| full name | Oxymetholone (17β-hydroxy-2-(hydroxymethylene)-17α-methyl-5α-androstan-3-one) |
| category | Anabolic & Androgenic |
| modality | steroid |
| formula | C21H32O3 |
| molar mass | 332.5 g/mol |
| cas | 434-07-1 |
laboratory handling
| storage | Analytical and reference material is typically stored as a dry solid at controlled room temperature protected from light; long-term standards are commonly held at −20 °C, sealed and desiccated. |
| solubility | Practically insoluble in water; soluble in ethanol, methanol, chloroform and DMSO. White to off-white crystalline solid. |
| co-studied with | This index does not describe combination use. Co-exposure to multiple 17α-alkylated oral androgens compounds hepatic injury risk, and oxymetholone is among the agents most frequently implicated in the cholestatic and peliosis case literature. |
Handling information describes laboratory practice for a research reagent. It is not a preparation guide for use in humans.
the receipts
5 citedLiverTox: Clinical and Research Information on Drug-Induced Liver Injury, NIDDK/NIH (NBK548931) · 2020 · official
LiverTox, NIDDK/NIH (NBK569838) · 2020 · official
PubChem, National Library of Medicine · 2026 · official
Circulation · 2017 · observational
Case Reports in Gastroenterology / PMC5043289 · 2016 · reviews
others in Anabolic & Androgenic