International Real-world study on osilodrostat efficacy and safety in adrenal Cushing syndrome - PubMed
5 hours ago
- #Cushing syndrome
- #adrenal adenoma
- #osilodrostat
- Objective: Assess efficacy and safety of osilodrostat in adrenal Cushing syndrome (CS).
- Methods: International study with patients treated with osilodrostat; safety evaluation for all, efficacy for those treated >4 weeks.
- Results: 28 patients enrolled (16 adrenocortical carcinoma, 12 benign disease). 66.7% responders (>4 weeks), increasing to 87.5% (>12 weeks).
- Non-first-line therapy (OR 15.0, P=0.010) predicted response.
- Significant decrease in systolic blood pressure and body weight (P<0.05).
- 9 patients had adverse events; 56% led to discontinuation.
- Conclusions: Osilodrostat controls hypercortisolism effectively, especially after 12 weeks, improving blood pressure and weight.
- Higher response rate in patients previously treated with other steroidogenesis inhibitors.