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 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 12.8mg (year 1) to 18.9mg (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 >3x 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.