Long-term safety and efficacy of subcutaneous pasireotide in patients with Cushing's disease: interim results from a long-term real-world evidence study.

Abstract

PURPOSE:

Clinical trials have demonstrated the favorable efficacy/safety profile of pasireotide in patients with Cushing's disease (CD). We report interim long-term results of an ongoing real-world evidence study of subcutaneous pasireotide in patients with CD.

METHODS:

Adults with CD receiving pasireotide, initiated before (prior-use) or at study entry (new-use), were monitored for ≤ 3 years during a multicenter observational study ( http://clinicaltrials.gov identifier NCT02310269). Primary objective was to assess long-term safety of pasireotide alone or with other CD therapies.

RESULTS:

At the time of this interim analysis, 127 patients had received pasireotide (new-use, n = 31; prior-use, n = 96). Eight patients had completed the 3-year observation period, 53 were ongoing, and 66 had discontinued. Among 31 new-use and 92 prior-use patients with ≥ 1 safety assessment, respectively: 24 (77%) and 37 (40%) had drug-related adverse events (AEs); 7 (23%) and 10 (11%) had serious drug-related AEs. Most common drug-related AEs were nausea (14%), hyperglycemia (11%) and diarrhea (11%); these were more frequently reported in new users and mostly of mild-to-moderate severity. 14 (45%) new-use and 15 (16%) prior-use patients experienced hyperglycemia-related AEs. Mean urinary free cortisol (mUFC) was within normal range at baseline and months 1, 12 and 24, respectively, in: 1/16 (6%), 9/18 (50%), 1/3 (33%) and 0/0 new users; 28/43 (65%), 15/27 (56%), 27/33 (82%) and 12/19 (63%) prior users.

CONCLUSIONS:

Pasireotide is well tolerated and provides sustained reductions in mUFC during real-world treatment of CD. The lower rate of hyperglycemia-related AEs in prior users suggests that hyperglycemia tends not to deteriorate if effectively managed soon after onset.

CLINICAL TRIAL REGISTRATION NUMBER:

NCT02310269.

• Authors: Luca Manetti, Timo Deutschbein, Jochen Schopohl, Kevin C J Yuen, Michael Roughton, Ulrike Kriemler-Krahn, Libuse Tauchmanova, Ricardo Maamari, Carla Giordano
• Journal: Pituitary (Pituitary) Vol. 22 Issue 5 Pg. 542-551 (Oct 2019) ISSN: 1573-7403 [Electronic] United States
• PMID: 31440946 (Publication Type: Clinical Trial, Journal Article)

Chemical References

• Somatostatin
• pasireotide

Topics

• Adult
• Female
• Humans
• Hyperglycemia (physiopathology)
• Male
• Middle Aged
• Multicenter Studies as Topic
• Pituitary ACTH Hypersecretion (drug therapy, physiopathology)
• Somatostatin (adverse effects, analogs & derivatives, therapeutic use)
• Treatment Outcome