Adrenal disease

Prospective study of metyrapone in endogenous Cushing's syndrome (PROMPT)

Eur J EndocrinolPublished Prospective single-arm study

An erratum to this article was published (Eur J Endocrinol, 28 Aug 2026 ↗)

Authors' conclusion

Metyrapone is a safe and effective treatment for endogenous CS.

Study context

Population
Adults with endogenous Cushing's syndrome (CS) (unsuitable for or uncontrolled after surgery) and 3 urinary free cortisol (UFC) concentrations each ≥1.5-fold upper limit of normal (ULN)49 evaluable
Participants
50 adults
Outcome
Normalization of mUFC
Follow-up
Week 12
Methodological detailsinternational, open-label
  • International
  • Open-label

Main finding

As reported in the abstract

Primary endpoint met (mUFC normalization), % of participants

46.9% (95% CI 32.5%-61.7%) (95% CI)

Observational data: associations, not effects.

Further findings

Normalization of mUFC · week 24

Efficacy maintained, % of participants
52.5%; 95% CI 37.5%-67.1% (95% CI)
  • 40 evaluated at week 24

Normalization of mUFC · week 36

Efficacy maintained, % of participants
48.6%; 95% CI 33.0%-64.4% (95% CI)
  • 35 evaluated at week 36

Responders (normalization or ≥50% decrease of baseline mUFC) · weeks 12, 24 and 36

Responder rate, week 12
80.9%
Responder rate, week 24
77.5%
Responder rate, week 36
71.4%

Clinical features and quality of life (QoL)

Clinical features and QoL improved

Safety

Mild-to-moderate adverse events (AEs), mostly during first 12 weeks
47 (94%)
Nausea
38%
Fatigue
26%
Headache
22%
Severe AEs
8
Reversible adrenal insufficiency during titration
6

Parts of this Brief are withheld: the automatic checks could not confirm them against the abstract.

Original publication

Prospective study of metyrapone in endogenous Cushing's syndrome (PROMPT). Eur J Endocrinol. Published 12 September 2025. doi:10.1093/ejendo/lvaf181

Composed by AI from the published abstract and checked against it. How Briefs are made →

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