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Endocrine Abstracts (2020) 70 AEP661 | DOI: 10.1530/endoabs.70.AEP661

ECE2020 Audio ePoster Presentations Pituitary and Neuroendocrinology (217 abstracts)

Metyrapone treatment in endogenous Cushing’s syndrome. Results from a prospective multicenter, open-label, phase III/IV study: Prompt

Lynnette Nieman 1 , Marco Boscaro 2 , Scaroni Carla 2 , Timo Deutschbein 3 , Emese Mezosi 4 , Natacha Driessens 5 , Carmen Emanuela Georgescu 6 , Alicja Hubalewska-Dydejczyk 7 , Dilek Berker 8 , Barbara Jarzab 9 , Dominique Maiter 10 , Martin Reincke 11 , Paola Loli 12 , Benedetta Zampetti 12 , Aysegul Atmaca 13 , Corin Badiu 14 , Albert Beckers 15 , Marek Bolanowski 16 , Francesco Cavagnini 17 , Nicole Unger 18 , Roberta Giordano 19 , Felicia Hanzu 20 , Massimo Terzolo 21 , Martine Bostnavaron 22 , Pauline Marsault 22 & Miklos Toth 23

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1National Institute of Diabetes and Digestive and Kidney Diseases, Endocrinology Department, BETHESDA, United States; 2University of Padova -, Endocrinology Department, Padova, Italy; 3University Hospital, University of Würzburg, Department of Internal Medicine, Division of Endocrinology and Diabetes, Würzburg, Germany; 4University Medical School, Department of Internal Medicine, Pecs, Hungary; 5CUB Hôpital Erasme, Université Libre de Bruxelles, Endocrinology department, Bruxelles, Belgium; 6Iuliu Hatieganu University of Medicine and Pharmacy & Cluj County Emergency Hospital, Endocrinology Department, Cluj-Napoca, Romania; 7Jagiellonian University, Medical College, Endocrinology Department, Krakow, Poland; 8Numune Training and Research Hospital, Ankara, Endocrinology Department, Altindag – Ankara, Turkey; 9Maria Sklodowska-Curie National Research Institute of Oncology, Department of Nuclear Medicine and Endocrine Oncology, Gliwice Branch, Gliwice, Poland; 10University Hospital Saint Luc, Endocrinologie et nutrition, Bruxelles, Belgium; 11University Clinic, Münich, Endocrinology department, Müunich, Germany; 12Niguarda Hospital, Endocrinology Department, Milan, Italy; 13Ondokuz Mayıs University, Endocrinology Department, Samsun, Turkey; 14C.I Parhon’, National Institute of Endocrinology, University of Medicine and Pharmacy, Endocrinology Department, Bucharest, Romania; 15University Hospital, Liege, Endocrinology Department, Liege, Belgium; 16Medical University, Department of Endocrinology, Diabetes and Isotope Therapy, Wroclaw, Poland; 17Hospital San Luca, Endocrinology Department, Milan, Italy; 18University Hospital Essen, Department of Endocrinology and Metabolism, Essen, Germany; 19University of Turin, Endocrinology Department, Turin, Italy; 20Hospital clinic, Endocrinology Department, Barcelona, Spain; 21San Luigi Gonzaga Hospital, Department of Clinical and Biological Sciences, Orbassano, Italy; 22HRA-Pharma Rare diseases, Medical affair, Châtillon, France; 23Semmelweis University, 2nd Department of Internal Medicine, Budapest, Hungary

Metyrapone treatment in endogenous Cushing’s syndrome. Results from a prospective multicenter, open-label, phase III/IV study: PROMPT

Background: Metyrapone is a steroidogenesis inhibitor approved in Europe for the treatment of endogenous Cushing’s syndrome (CS) based on observational retrospective studies published over more than 50 years. We present data from the first prospective study designed to confirm metyrapone efficacy and good tolerance in patients with CS.

Methods: This single arm, open-label, multicenter, international trial enrolled 50 patients with CS who had three baseline 24 hours urine free cortisol (UFC) values at least 50% above the upper limit of normal (ULN = 165 nmol/24 h). Metyrapone was titrated over 12 weeks (W12) to achieve normal urine (mean of 3 values, mUFC) and serum cortisol levels. Patients whose mUFC did not exceed 2.5-fold the ULN could enter a 6-month extension period. The primary efficacy endpoint was the proportion of patients with mUFC ≤ ULN at W12 assessed in a central laboratory using LC-MS/MS. The most important secondary endpoint was a mUFC decrease of ≥ 50% at W12.

Results: At baseline, mean age was 47 years, mean mUFC (s.d.) was 1042 (1337) nmol/24 h and median mUFC (range) was 570 (291–8476) nmol/24 h (3.5 × ULN). Diabetes mellitus (65%) and hypertension (51%) were the most common comorbidities at baseline. At W12: 48% (23 of 48 patients) met the primary endpoint. Another 40% (19 of 48 patients) had mUFC ≤ 2 × ULN at W12. Median percentage decrease in mUFC from baseline to W12 was –74%. Secondary endpoint was met by 81% of patients who had a mUFC decrease of 50%. Final median metyrapone dose was 1500 (250; 5500) mg/day at W12. At W12, circulating cholesterol, HbA1C and fasting glucose improved with mean decrease of 12%, 3% and 6% respectively and median systolic and diastolic blood pressure also decreased by 3.4 and 3.5 mmHg respectively. At W12, ACTH increased 20% (+ 9 ng/l) from baseline. Twenty six (52%) patients experienced mild to moderate related adverse events (AEs). One patient discontinued before W12 because of an unrelated SAE on day 2 (pneumonia with septic shock). The most common AEs were nausea (24%), decreased appetite (18%), fatigue (14%), headache (10%), peripheral edema (6.0%), hypokalemia (6.0%) and hypertension (6.0%). Reversible adrenal insufficiency occurred in 6 (12%) patients.

Conclusions: This prospective study in patients with CS confirms that metyrapone effectively lowers UFC levels with a tolerability profile similar to the previously reported safety profile.

Volume 70

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22nd European Congress of Endocrinology

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05 Sep 2020 - 09 Sep 2020

European Society of Endocrinology 

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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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