Summary

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• This summary is based on the review of two systematic review(s)/meta-analysis(es). ^[1-2]
• Osilodrostat significantly increased the likelihood of achieving normal 24-hour urine free cortisol (UFC) levels in patients with Cushing's syndrome, with an odds ratio (OR) of 21.94 (95% CI (confidence interval): 8.53-56.43), indicating a strong effect (P < 0.00001).
• The proportion of disease control in Cushing’s disease for patients treated with osilodrostat was 66.4% (95% CI: 57.9-74.3), which was higher than for cabergoline (35%), pasireotide (44%), and ketoconazole (41%), while being similar to metyrapone (66%).
• The odds of experiencing adverse events with osilodrostat were similar to placebo (OR 1.35, 95% CI: 0.52-3.53, P = 0.54), as were the rates of serious adverse events (OR 1.32, 95% CI: 0.30-5.79, P = 0.72). However, osilodrostat had a higher incidence of nausea (OR 4.25, 95% CI: 1.26-14.30, P = 0.02) and arthralgia (OR 6.54, 95% CI: 1.64-26.13, P = 0.008) compared to placebo.
• There were no significant safety concerns related to other specific adverse events, such as adrenal insufficiency, headache, or hyperandrogenism.
• Studies on osilodrostat involved patients with Cushing's syndrome and Cushing's disease, but no specific subgroups (e.g., based on age, gender, or comorbid conditions) were explicitly highlighted regarding differences in effectiveness or safety outcomes.