Pegvisomant treatment in acromegaly in clinical practice: Final results of the French ACROSTUDY (312 patients).

Abstract Objective We report the final analysis of the French ACROSTUDY, using data revised and enriched since the 2013 interim analysis. Our objective was to validate the use of pegvisomant (PEGV) in the treatment of acromegaly and to determine efficacy and safety. Patients and methods Patients with acromegaly treated with PEGV and followed up for at least 5 years were included. Eighty-eight investigators from 62 clinical centers in France included patients from April 2007 to April 2014. PEGV dose and administration frequency were determined by the physicians, based on their clinical evaluation and local habits. No additional examinations beyond those performed in normal follow-up were required. Minimum recommended follow-up included check-ups at treatment initiation, 6 months, 12 months and then annually. Results In total, 312 patients were enrolled. Mean age was 46.1 ± 14.3 years at introduction of PEGV. Median PEGV treatment duration was 6.3 years and median follow-up was 5.6 years. Median dose at initiation was 10 mg/day. The percentages of patients with IGF-1 ≤ ULN (upper limit of normal) were 10% (n = 300) at baseline, 54% at 6 months (n = 278), and 61.7% (n = 253) at 2 years, then stabilizing at 64.4% (n = 180) at 5 years. Mean PEGV dose was 17.4 ± 11.7 mg in patients with controlled disease versus 21.1 ± 17.3 mg in those without control at 5 years. At 5 years, 21.8% of patients (54/248) were receiving >30 mg PEGV per day. In patients with at least one pituitary imaging procedure during the 5-year follow-up (n = 292), the most recent image showed stable tumor volume in 212 subjects (72.6%), increased volume in 13 (4.5%), and decreased volume in 30 (10.3%). No PEGV treatments were permanently discontinued due to transaminase elevation. There were no cases of liver failure. Conclusion The French ACROSTUDY showed normalization of IGF-1 levels in 64.4% of a real-life cohort of patients, mostly with uncontrolled disease despite multiple prior therapies. Long-term follow-up showed a sustained effectiveness and good long-term safety.