Real-World Effectiveness and Safety of Osilodrostat in Cushing’s Syndrome: A Retrospective Phase IV Study
Authors' conclusion
Osilodrostat is effective, generally well tolerated in routine clinical practice, representing a valuable therapy for CS management, able to achieve rapid and sustained hormonal control, significant metabolic, cardiovascular, clinical and QoL improvements with a well-tolerated safety profile.
Study context
- Population
- Patients with Cushing's syndrome (CS) (74% pituitary, 16% adrenal, 10% ectopic), 11 Italian endocrine referral centers
- Participants
- 100 patients
- Outcome
- Percentage of patients with UFC≤ULN
- Follow-up
- Last observation
Methodological detailsmulticentre, phase IV, 11 Italian endocrine referral centers
- Multicentre
- Phase IV
- 11 Italian endocrine referral centers
Main finding
As reported in the abstract
Patients with normal UFC levels at last observation
81%
Observational data: associations, not effects.
Secondary outcomes
Normal UFC at least once · within study period
- Patients with normal UFC at least once within study period
- 92%
Normal UFC, excluding 12 patients with normal baseline UFC
- Normal UFC at last observation
- 78.4%
- Normal UFC at least once within study period
- 90.9%
Other hormonal and clinical outcomes
- Significant reductions in late-night salivary cortisol and morning serum cortisol levels
- Improvements in anthropometric parameters, blood pressure, glucose metabolism, clinical signs and QoL
- Hormonal control consistent across CS etiologies
Safety
- AEs, mostly mild-to-moderate
- 29%
- Adrenal insufficiency (most common AE)
- 20%
- Safety profiles consistent across CS etiologies
Parts of this Brief are withheld: the automatic checks could not confirm them against the abstract.
Original publication
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