INTRODUCTION: Cushing’s disease (CD) significantly impacts signs and symptoms, as well as health-related quality of life (HRQoL). Pasireotide demonstrated efficacy in the Phase III study of 162 patients with active CD randomized to pasireotide 600 or 900 µg twice daily.1 This post-hoc analysis assessed the effect of pasireotide on cortisol, clinical signs and symptoms (diastolic blood pressure, weight, BMI, and depression measured by Beck Depression Inventory-II score) and patient-reported outcomes in a subset of 78 patients who completed 12 months of therapy in this trial.
METHODS: Treatment groups were combined for the analysis. Urinary free cortisol (UFC), CD signs and symptoms, and HRQoL (CushingQoL2) were evaluated. The minimal important difference (MID) was validated to be 10.1 points on the CushingQoL scale. The correlations between changes in CushingQOL and in UFC and signs/symptoms were computed. Correlation coefficients of 0.1, 0.1–0.5 and >0.5 are considered weak, moderate and strong associations, respectively.
RESULTS: At 6 months, 37% of patients normalized UFC. 80% of patients either had a reduction in diastolic blood pressure =5 mmHg (58%), decrease in weight =7% (42%) or had improvement =MID on CushingQoL (46%) (mean±SD, 11.1±15.2). Improvements in symptoms and HRQoL were maintained at 12 months. Improvement in CushingQoL was correlated (P<0.01) with changes in UFC at 12 months (r=–0.40), and at 6 and 12 months with BMI (r=–0.39; –0.31), weight (r=–0.41; –0.32) and depression (r=–0.54; –0.59).
CONCLUSIONS: Pasireotide leads to meaningful improvements in HRQoL in patients with CD. Improvements in UFC, weight and depression are associated with improvements in HRQoL. The 11.1 improvement in CushingQoL score observed in this study at 6 months was comparable to the 10-point difference historically reported for patients with uncontrolled versus controlled CD by other modalities.2 References: Colao A et al. 2012, 2Webb SM et al. 2008.
