Long-term treatment with pegvisomant: observations from 2090 acromegaly patients in ACROSTUDY. Issue 6 (December 2018)

• Record Type: Journal Article
• Authors: Buchfelder, Michael; van der Lely, Aart-Jan; Biller, Beverly M K; Webb, Susan M; Brue, Thierry; Strasburger, Christian J; Ghigo, Ezio; Camacho-Hubner, Cecilia; Pan, Kaijie; Lavenberg, Joanne; Jönsson, Peter; Hey-Hadavi, Juliana H
• Is Part Of: European journal of endocrinology. Volume 179:Issue 6(2018)
• Page Start: 419
• Page End: 427
• Publication Date: 2018-12
• Subjects: Endocrinology -- Periodicals; 616.4005
• DOI: 10.1530/EJE-18-0616
• Languages: English
• ISSNs: 0804-4643

Abstract

Abstract : Objectives: ACROSTUDY is an international, non-interventional study of acromegaly patients treated with pegvisomant (PEGV), a growth hormone receptor antagonist and has been conducted since 2004 in 15 countries to study the long-term safety and efficacy of PEGV. This report comprises the second interim analysis of 2090 patients as of May 12, 2016. Methods: Descriptive analyses of safety, pituitary imaging and outcomes on PEGV treatment up to 12 years were performed. Results: Prior to starting PEGV, 96% of patients had reported surgery, radiation, medical therapy or any combinations of those. At start of PEGV, 89% of patients had IGFI levels above the upper limit of normal (ULN). The percentage of patients with normal IGFI levels increased from 53% at year 1 to 73% at year 10, and the average daily dose of PEGV increased from 12.8 mg (year 1) to 18.9 mg (year 10). A total of 4832 adverse events (AEs) were reported in 1137 patients (54.4%), of which 570 were considered treatment related in 337 patients (16.1%). Serious AEs were reported in 22% of patients, of which 2.3% were considered treatment related. Locally reported MRIs showed most patients (72.2%) had no change in tumor size relative to the prior scan; 16.8% had a decrease, 6.8% an increase and 4.3% both. In patients with normal liver tests at PEGV start, an ALT or AST elevation of >3× ULN at any time point during their follow-up was reported in 3%. Conclusions: This second interim analysis confirms that long-term use of PEGV is an effective and safe treatment in patients with acromegaly.