Long-term treatment with pegvisomant: Observations from 2090 acromegaly patients in ACROSTUDY

Michael Buchfelder, Aart Jan Van Der Lely, Beverly M.K. Biller, Susan M. Webb, Thierry Brue, Christian J. Strasburger, Ezio Ghigo, Cecilia Camacho-Hubner, Kaijie Pan, Joanne Lavenberg, Peter Jönsson, Juliana H. Hey-Hadavi

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60 Citations (Scopus)


© 2018 European Society of Endocrinology Printed in Great Britain 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.
Original languageEnglish
Pages (from-to)419-427
JournalEuropean Journal of Endocrinology
Publication statusPublished - 1 Dec 2018


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