Real-World Experience with Pasireotide-LAR in Cushing’s Disease: Single-Center 12-Month Observational Study

Abstract

Background/Objectives: Pasireotide-LAR represents a novel therapeutic option for patients with Cushing’s disease (CD). Its efficacy and safety were assessed in clinical trials; however, the real-world evidence is still scarce. Methods: The study aimed to evaluate the impact of 12-month pasireotide-LAR therapy on disease control, glucose metabolism, lipid profiles, and adverse effects in a real-life setting. We retrospectively studied prospectively collected data of patients with persistent or recurrent CD administered with pasireotide-LAR in a single pituitary center. Results: Mean urinary free cortisol (mUFC) showed a sustained decrease from baseline, with the most pronounced decrease in the first 3 months of therapy (p = 0.007). The analysis of mean late-night salivary cortisol showed fluctuations over time, with the largest mean reduction in mLNSC at 3 months. During the therapy, an improvement in blood pressure control was observed, with a significant decrease in systolic blood pressure during the first 6 months of treatment (p = 0.005). Hyperglycemia was the most common adverse effect. Fasting plasma glucose and glycated hemoglobin (HbA1c) showed a gradual increase during pasireotide-LAR treatment, with the HbA1c significantly increasing at the last follow-up (p = 0.04). Conclusions: Pasireotide-LAR is an effective alternative treatment in selected patients with CD. Pasireotide-LAR is overall safe and well tolerated, with hyperglycemia being the most common but manageable adverse event.