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

<b>Background/Objectives</b>: 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. <b>Methods</b>: 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. <b>Results:</b> Mean urinary free cortisol (mUFC) showed a sustained decrease from baseline, with the most pronounced decrease in the first 3 months of therapy (<i>p</i> = 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 (<i>p</i> = 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 (<i>p</i> = 0.04). <b>Conclusions</b>: 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.

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Publication Details

Journal
Journal of Clinical Medicine
Published
2025-04-18
DOI
https://doi.org/10.3390/jcm14082794
Citations
3
Primary Topic
Pituitary Gland Disorders and Treatments
Type
article
Field-Weighted Citation Impact
3.88

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article

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

Przemysław Witek, Wioleta Respondek, Łukasz Działach
3 citations
Journal of Clinical Medicine
Pituitary Gland Disorders and Treatments
3.88
article

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

Przemysław Witek, Wioleta Respondek, Łukasz Działach
article en
3 citations

Abstract

<b>Background/Objectives</b>: 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. <b>Methods</b>: 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. <b>Results:</b> Mean urinary free cortisol (mUFC) showed a sustained decrease from baseline, with the most pronounced decrease in the first 3 months of therapy (<i>p</i> = 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 (<i>p</i> = 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 (<i>p</i> = 0.04). <b>Conclusions</b>: 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.

Journal of Clinical MedicineVol. 14(8)
Medical University of Warsaw (PL)
Warszawski Uniwersytet Medyczny, Uniwersytet Warszawski
Good health and well-being
Openalex Percentile: Top 7%
Pituitary Gland Disorders and Treatments
3.88
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