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Endocrine Abstracts (2022) 81 OC7.3 | DOI: 10.1530/endoabs.81.OC7.3


1Université Paris-Saclay – Assistance Publique-Hôpitaux de Paris , Endocrinology, Paris, France; 2Centre Hospitalier Universitaire Bordeaux, Bordeaux, France, Endocrinology, France; 3Centre Hospitalier Universitaire Nantes, Nantes, France, Endocrinology, France; 4Centre Hospitalier Régional Universitaire Nancy, Nancy, Endocrinology, France; 5Centre Hospitalier Régional Universitaire Lille, Endocrinology, France; 6Le Centre Hospitalier du Mans, Endocrinology, France; 7Centre Hospitalier Universitaire Caen, Endocrinology, France; 8Centre Hospitalier Universitaire Nantes, Endocrinology, France; 9Centre Hospitalier Universitaire Montpellier, Endocrinology, France; 10Hospital Center Universitaire, Clermont-Ferrand, France; 11Centre Hospitalier Régional Universitaire Lille, Endocrinology, Lille, France; 12Centre Hospitalier Universitaire Reims, Endocrinology, France; 13Centre Hospitalier Universitaire Reims, Reims, France; 14Centre Hospitalier Régional Universitaire Strasbourg, Endocrinology, France; 15Centre Hospitalier de Chambéry - Hôpital Savoie, Endocrinology, France; 16Centre Hospitalier Universitaire Dijon, Endocrinology, France; 17Institut Gustave Roussy, Endocrinology, France; 18Centre Hospitalier Universitaire Marseille, Endocrinology, France; 19Centre Hospitalier Universitaire Lyon, Endocrinology, France; 20CHU Grenoble, Endocrinology, France; 21Centre Hospitalier Universitaire Bordeaux, Endocrinology, France


Background: Osilodrostat is a potent oral inhibitor of the adrenal enzymes aldosterone synthase and 11b-hydroxylase and decreases glucocorticoid and mineralocorticoid production and secretion. Phase 2 and 3 studies from the osilodrostat clinical trial programme have demonstrated the drug’s efficacy and safety in patients with Cushing’s disease. Osilodrostat received European Marketing Authorization (MA) for the treatment of Cushing’s syndrome (CS) in adults.

Objective: Evaluate the use of osilodrostat for the treatment of CS in clinical practice in France (Autorisation Temporaire d’Utilisation [ATU], post-ATU, post-MA; authorization IDRCB2021A0140140).

Methods: This multicentre analysis included patients with CS who were treated with osilodrostat between 2019 and 2021. Causes of CS, therapeutic approaches, dosages, and efficacy and safety of osilodrostat were analysed in patients where data was available.

Results: Patients (n=107) with CS aged 11–85 years were analysed; 68 patients were female and 39 were male. At diagnosis, urinary free cortisol (UFC; median ± standard deviation (SD) [range; n]) was 135 μg/24 h ±1703 (10–27188; n=79). Causes of CS in these patients include ACTH-dependent (Cushing’s disease [n=57]; ectopic [n=28]; uncertain [n=5]) and ACTH-independent (adrenocortical carcinoma [n=9]; macronodular adrenocortical hyperplasia [n=5]; adrenocortical adenoma [n=2]). UFC levels (median ± SD [range; n]) at the time of initiating osilodrostat therapy were 135 μg/24 h ±1703 (5–10000; n=78). Regarding therapeutic approaches (n/N, %), 17/87 patients (20%) received osilodrostat as first-line therapy, whereas 36 patients (41%) received it as second-line therapy. Methods of osilodrostat administration included: titration (59/95, 62%), block and replace (9/84, 11%) and titration followed by block and replace (33/85, 39%). The initial osilodrostat dose (median ± SD [range; n]) was 4.0 mg/day ±8.7 (1–60; n=96), whereas the maximum dose was 12.0 mg/day ±18 (1–80; n=103). UFC normalization with osilodrostat was achieved in 64/78 patients (82%). Improvements of clinical signs and symptoms were reported in 67/89 patients (75%), and improvement of comorbidities with osilodrostat was reported in 39/62 patients (63%). In terms of safety in patients treated with osilodrostat, adrenal insufficiency was reported in 30/91 patients (33%) and hyperandrogenism was reported in 6/54 patients (11%).

Conclusion: These findings from clinical practice confirm the efficacy of osilodrostat in patients with various aetiologies of CS, in agreement with the European MA. The main side effect observed was adrenal insufficiency, which was expected and related to the mechanism of action of osilodrostat. Use of the block and replace approach may prevent the development of adrenal insufficiency.

Volume 81

European Congress of Endocrinology 2022

Milan, Italy
21 May 2022 - 24 May 2022

European Society of Endocrinology 

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