Real-World Osilodrostat Effectiveness and Safety in Nonpituitary Cushing Syndrome

Area of Science:

• Endocrinology
• Pharmacology
• Clinical Medicine

Background:

• Limited data exists on osilodrostat for non-pituitary Cushing syndrome (CS).
• Most clinical trials focused on Cushing disease patients.

Purpose of the Study:

• To evaluate the effectiveness and safety of osilodrostat in real-world practice for non-pituitary CS in France.
• Assess osilodrostat's role in managing diverse non-pituitary CS etiologies.

Main Methods:

• Retrospective, observational, multicenter study (LINC 7; NCT05633953) in France.
• Included 103 adult non-pituitary CS patients (ectopic ACTH syndrome, adrenocortical carcinoma, adrenal adenoma, bilateral adrenal nodular disease).
• Data collected from 2019-2022, analyzing osilodrostat exposure, dosage, and efficacy (mean urinary free cortisol levels).

Main Results:

• Osilodrostat initiation led to cortisol reduction by week 4, sustained thereafter.
• 44.2% of patients achieved normalized mean urinary free cortisol (mUFC) by week 12.
• Common adverse events included adrenal insufficiency (28%) and hypokalemia (18%).

Conclusions:

• Osilodrostat demonstrates suitability as a treatment for endogenous Cushing syndrome arising from various non-pituitary sources.
• The study supports osilodrostat's use in real-world settings for non-pituitary CS management.