Efficacy and Safety of Osilodrostat in Paraneoplastic Cushing Syndrome: A Real-World Multicenter Study in France.

ABSTRACT:

Context

Prospective studies have demonstrated the efficacy of osilodrostat in Cushing disease. No study has evaluated osilodrostat in a series of patients with paraneoplastic Cushing syndrome/ectopic adrenocorticotropin syndrome (PNCS/EAS).

Objective

This work aimed to evaluate in France the real-world efficacy and safety of osilodrostat in patients with PNCS/EAS.

Methods

A total of 33 patients with PNCS/EAS with intense/severe hypercortisolism were involved in this retrospective, multicenter, real-world study. Patients received osilodrostat between May 2019 and March 2022 at a median initial dose (range) of 4 mg/day (1-60) and maximum dose, 20 mg/day (4-100), first under patient then cohort temporary authorizations and after marketing authorization. Regimens used tit

SUBMITTER: Dormoy A 

PROVIDER: S-EPMC10188310 | biostudies-literature | 2023 May

REPOSITORIES: biostudies-literature