ECE2020 Audio ePoster Presentations Pituitary and Neuroendocrinology (217 abstracts)
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 (
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.