Clinical Scorecard: Personalized Osilodrostat Therapy for Individuals with ACTH-Dependent Cushing’s Syndrome: Insights from Real-World Data

At a Glance

Key Highlights

• 92% of patients achieved a therapeutic effect at a median dose of 5 mg/day within 14 days.
• Morning cortisol normalized in 92% of cases and urinary free cortisol in 75%.
• Median clinical score decreased from 9 to 4, with muscle strength improvement from 50% to 75% of normal.
• Systolic/diastolic blood pressure decreased significantly, allowing reduction of antihypertensive therapy in most patients.
• Adverse events were mild to moderate, with fatigue and nausea reported in 34.6% of patients.

Guideline-Based Recommendations

Diagnosis

• Diagnosis of ACTH-dependent Cushing’s syndrome should include assessment of cortisol levels and imaging studies.

Management

• Osilodrostat is recommended as a second-line pharmacological therapy for patients with ACTH-dependent Cushing’s syndrome.

Monitoring & Follow-up

• Regular monitoring of cortisol levels and clinical symptoms is essential during osilodrostat therapy.

Risks

• Potential adverse events include fatigue, nausea, adrenal insufficiency, and dizziness.

Patient & Prescribing Data

26 adults with ACTH-dependent Cushing’s syndrome treated with osilodrostat.

Osilodrostat was introduced as first-line therapy in 12 patients, with a titration regimen used in 21 patients.

Clinical Best Practices

• Individualized dosing and titration of osilodrostat based on patient response.
• Close monitoring of blood pressure and glucose levels during treatment.

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