Background: In a large 12-month Phase III study, pasireotide led to rapid and sustained decreases in UFC and provided clinical benefit in patients with Cushings disease. Here, we report data following an open-label, open-ended extension.
Methods: 162 patients with persistent/recurrent or de novo Cushings disease were randomized in the core study. 58 patients with mean UFC≤ULN or clinical benefit at month 12 entered the extension. Dose titration was permitted at the investigators discretion (3001200 μg s.c. bid). Efficacy data are presented for those patients who attended the visit at month 60; safety data are presented up to study end for all randomized patients.
Results: 16 and seven patients were ongoing at months 60 and 72, respectively; maximum exposure was 76.6 months. For patients who reached month 60, percentage change in median UFC (range) from core baseline was −82.6% (−96.5% to 324.0%; n=16) and −81.8% (−94.9% to 3.4%; n=15) at months 12 and 60, respectively; of these patients, 10/16 and 11/16 had UFC≤ULN at months 12 and 60, respectively. Percentage change in median ACTH (range) was −34.7% (−72.0% to 66.7%; n=16) at month 12 and −8.3% (−68.0% to 100.0%; n=16) at month 60. Improvements in clinical signs, including systolic and diastolic blood pressure, weight, BMI, and total cholesterol level, were observed by month 6 and were sustained up to month 60. The most common AEs reported from core baseline until study end were diarrhoea (58.6%), nausea (53.7%), hyperglycaemia (41.4%), and cholelithiasis (32.7%); diabetes mellitus was reported in 22.2% of patients.
Conclusions: Initial reductions in UFC and improvements in clinical signs were maintained over 5 years of pasireotide treatment. No new safety signals were reported during long-term treatment. Results from this study suggest that pasireotide may be effective in the long-term treatment of Cushings disease.
Disclosure: This work was supported by Novartis.
16 - 20 May 2015
European Society of Endocrinology