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.

Keywords: Cushing’s disease; cortisol; hyperglycemia; pasireotide; pituitary tumor.

Figure 1

Mean and SD changes in morning serum cortisol (A), midnight serum cortisol (B), ACTH (C), mUFC (D), and mLNSC (E) throughout study. * is provided when significant difference (p < 0.05) compared to baseline was found, and ** when p < 0.01. Abbreviations: ACTH, adrenocorticotropin; LOV, last observed value; mLNSC, mean late-night salivary cortisol; m, months; mean late-night salivary cortisol; mUFC, mean urinary free cortisol.

Figure 2

Mean and SD changes in weight (A), BMI (B), SBP (C), and DBP (D) throughout study. * is provided when significant difference (p < 0.05) compared to baseline was found, and ** when p < 0.01. Abbreviations: BMI, body mass index; DBP, diastolic blood pressure; LOV, last observed value; SBP, systolic blood pressure.

Figure 3

Mean and SD changes in FPG (A) and HbA1c (B) throughout study. * is provided when significant difference (p < 0.05) compared to baseline was found. Abbreviations: FPG, fasting plasma glucose; HbA1c, glycated hemoglobin; LOV, last observed value.