Evidence map›Paper›PMID 40966724›Full record

Trial reportEuropean journal of endocrinology2025

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

Lynnette K Nieman, Marco Boscaro, Carla Scaroni, Timo Deutschbein, Emese Mezôsi, Natacha Driessens, Carmen E Georgescu, Marcin Motyka, Alicja Hubalewska-Dydejczyk, Barbara Jarzab and 17 more

Erratum issuedAbstract readMulticenter StudyClinical Trial
In one paragraph

Trial report in European journal of endocrinology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

6 citing papers in PubMed.

  1. Trial
  2. Review
  3. Article
  4. Review
  5. Article
  6. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

27 authors.

Lynnette K NiemanNational Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Building 10, CRC, 1 East, Rm 1-3140, 10 Center Dr, MSC 1109, Bethesda, MD 20892-1109, United States.ORCID 0000-0003-0534-8025
Marco BoscaroEndocrinology Unit, Department of Medicine, Hospital-University, Padova, 35128, Italy.ORCID 0000-0003-2596-1652
Carla ScaroniEndocrinology Unit, Department of Medicine, Hospital-University, Padova, 35128, Italy.ORCID 0000-0001-9396-3815
Timo DeutschbeinDepartment of Internal Medicine I, Division of Endocrinology and Diabetes, University Hospital, University of Würzburg, Würzburg, 97080, Germany.
Emese MezôsiDepartment of Internal Medicine, University Medical School, Pecs, 7624, Hungary.
Natacha DriessensHôpital Universitaire de Bruxelles Hôpital Erasme, Université Libre de Bruxelles, Brussels, 1070, Belgium.ORCID 0000-0002-5992-2486
Carmen E GeorgescuDepartment of Endocrinology, Iuliu Hatieganu University of Medicine and Pharmacy and Cluj Country Emergency Hospital, Cluj-Napoca, 400012, Romania.
Marcin MotykaDepartment of Endocrinology, Oncologic Endocrinology and Nuclear Medicine, University Hospital in Krakow, Krakow, 30-688, Poland.
Alicja Hubalewska-DydejczykEndocrinology Department, Jagiellonian University Medical College, Krakow, 30-688, Poland.
Barbara JarzabDepartment of Nuclear Medicine and Endocrine Oncology, Maria Sklodowska-Curie National Research Institute of Oncology, Gliwice Branch, Gliwice, 44-102, Poland.
Dominique MaiterDepartment of Endocrinology and Nutrition, UCLouvain Hospital Saint-Luc, Brussels, 1200, Belgium.ORCID 0000-0002-0918-637X
Martin ReinckeDepartment of Medicine 4, Klinikum der Universität, Ludwig-Maximilians-Universität München, Munich, 80336, Germany.ORCID 0000-0002-9817-9875
Paola LoliEndocrinology Department, Clinica San Carlo, Paderno Dugnano, Milan, 20037, Italy.
Benedetta ZampettiEndocrinology Unit, ASST Grande Ospedale Metropolitano Niguarda, Milan, 20162, Italy.
Aysegul AtmacaDepartment of Internal Medicine, Division of Endocrinology and Metabolism, Ondokuz Mayis University, Samsun, 55200, Turkey.
Corin BadiuDepartment of Endocrinology IV, "C.I Parhon", National Institute of Endocrinology, "D. Davila" University of Medicine and Pharmacy, Bucharest, 011863, Romania.ORCID 0000-0002-8087-8125
Albert BeckersEndocrinology Department, University Hospital, Liege, 4000, Belgium.
Marek BolanowskiDepartment of Endocrinology and Internal Medicine, Medical University Wroclaw, Wroclaw, 50-367, Poland.
Francesco CavagniniEndocrinology Clinic, Istituto Auxologico Italiano, Milan, 20100, Italy.
Nicole UngerDepartment of Endocrinology, Diabetes and Metabolism, University Hospital Essen, Essen, 45127, Germany.
Roberta GiordanoDepartment of Biological and Clinical Sciences, University of Turin, Turin,10100, Italy.ORCID 0000-0001-8713-3532
Felicia Alexandra HanzuEndocrinology Department, Hospital Clinic, University of Barcelona, Barcelona, 080336, Spain.ORCID 0000-0002-2399-4023
Massimo TerzoloDepartment of Clinical and Biological Sciences, Internal Medicine, San Luigi Gonzaga Hospital, Orbassano, 10043, Italy.
Myriam Bou NaderEsteve, Montrouge, 92120, France.
Ninet SinaiiBiostatistics and Clinical Epidemiology Service, National Institutes of Health Clinical Center, National Institute of Health, Bethesda, MD 20892, United States.ORCID 0000-0003-4045-1386
Judit TőkeDepartment of Internal Medicine and Oncology, Semmelweis University, Budapest, H-1083, Hungary.
Miklós TóthDepartment of Internal Medicine and Oncology, Semmelweis University, Budapest, H-1083, Hungary.

Funding

The differential diagnosis and treatment of Cushing's syndromeZIADK075122 · NIDDK · NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES · PI NIEMAN, LYNNETTE · 2016 to 2025
$4.0M
ESTEVEHRA PharmaIntramural NIH HHS ZIA DK075122
6 · The paper itself

Abstract

objectiveWe evaluated the safety and efficacy of metyrapone treatment for Cushing's syndrome (CS).

designInternational, prospective, single-arm, open-label study.

methodsFifty adults with endogenous CS (either unsuitable for or uncontrolled after surgery) and 3 urinary free cortisol (UFC) concentrations each ≥1.5-fold the upper limit of normal (ULN) were enrolled. After 12 weeks of metyrapone titration, participants with mean 24 h UFC (mUFC) ≤ 2-fold ULN could enter a 24-week extension phase. Safety was assessed, and doses adjusted at weeks 1-5, 8, 12, and 24. Pre-defined endpoints included normalization of mUFC at weeks 12 (primary), 24, and 36, and proportion of "responders" (normalization or ≥50% decrease of baseline mUFC), time to eucortisolemia, salivary cortisol day-curve, and quality of life (QoL).

resultsOf the 49 evaluable participants, 47 completed the 12-week visit; 40 were evaluated at week 24 and 35 at week 36. The primary endpoint was met in 46.9% of participants (95% CI 32.5%-61.7%), with efficacy maintained at week 24 (52.5%; 95% CI 37.5%-67.1%) and week 36 (48.6%; 95% CI 33.0%-64.4%). The responder rates were 80.9%, 77.5%, and 71.4% at weeks 12, 24, and 36, respectively. Forty-seven participants (94%) developed mild-to-moderate adverse events (AEs), mostly during the first 12 weeks and most commonly nausea (38%), fatigue (26%), and headache (22%); 8 experienced severe AEs. Six participants developed reversible adrenal insufficiency during titration. Clinical features and QoL improved.

conclusionMetyrapone is a safe and effective treatment for endogenous CS.

Indexed as

Cushing SyndromeMetyraponeAdultAgedFemaleHumansHydrocortisoneMaleMiddle AgedProspective StudiesQuality of LifeTreatment OutcomeYoung AdultHydrocortisoneMetyraponeCushing's syndromelate-night salivary cortisolmetyraponeurinary free cortisol

Identifiers

PMID40966724
PMCPMC13187941

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.