Long-term safety and efficacy of pegvisomant monotherapy for acromegaly: final data from the full ACROSTUDY cohort
Yuen KC, Vila G, Bernabeu I, Buchfelder M, Freda P, Henocque R, Torre DL, Maffei P, Strasburger CJ, Neggers S, Brue T (2026)
Publication Type: Journal article
Publication year: 2026
Journal
Book Volume: 29
Article Number: 71
Journal Issue: 3
DOI: 10.1007/s11102-026-01673-x
Abstract
Purpose: ACROSTUDY was an international, non-interventional, cross-sectional study that evaluated the long-term safety and treatment outcomes of pegvisomant treatment (monotherapy or combination) in patients with acromegaly in “real-world” clinical practice. This study aimed to characterize the long-term safety and efficacy of pegvisomant monotherapy in ACROSTUDY. Methods: Patients with acromegaly aged > 18 years (at ACROSTUDY enrollment) who had received ≥ 1 dose of pegvisomant as monotherapy were included in the monotherapy cohort. Clinical assessments included adverse events (AEs), comorbidities, pituitary imaging, liver function tests, and insulin-like growth factor-1 (IGF-I) serum measurements. Results: Patients with acromegaly (N = 1464) received pegvisomant monotherapy for a median (min, max) of 6.0 (0.0, 19.3) years and were followed in ACROSTUDY for a mean of 7.6 years. The rates of AEs (all-causality) and serious AEs were 49% (n = 717/1464) and 19.3% (n = 282/1464), respectively. The most common treatment-related AEs were IGF-I increased (1.8%), lipohypertrophy (1.2%), transaminases increased (1.2%), and IGF-I decreased (1.1%). There were 31 (2.1%) deaths and none were related to treatment. Magnetic resonance imaging findings at study sites showed 7.2% (n = 90/1248) of patients had increased tumor size, whereas 70.6% (n = 881/1248) of patients had change in tumor size. At the last observation, the serum IGF-I normalization rate was 62.3% (n = 756/1213) and the median (min, max) daily pegvisomant dose was 15.0 (0, 60) mg/day. Conclusions: In this largest and longest real-world study of pegvisomant monotherapy in patients with acromegaly to date, pegvisomant demonstrated a favorable safety profile and was effective at normalizing and maintaining IGF-I levels in the majority of patients.
Authors with CRIS profile
Involved external institutions
University of Arizona
United States (USA) (US)
Medizinische Universität Wien
Austria (AT)
Columbia University Irving Medical Center (CUIMC)
United States (USA) (US)
Pfizer France
France (FR)
Pfizer, Inc.
United States (USA) (US)
University of Padua / Universita degli Studi di Padova
Italy (IT)
Charité - Universitätsmedizin Berlin
Germany (DE)
Erasmus University Medical Center (MC)
Netherlands (NL)
Marseille Medical Genetics (MMG)
France (FR)
United States (USA) (US)
Medizinische Universität Wien
Austria (AT)
Columbia University Irving Medical Center (CUIMC)
United States (USA) (US)
Pfizer France
France (FR)
Pfizer, Inc.
United States (USA) (US)
University of Padua / Universita degli Studi di Padova
Italy (IT)
Charité - Universitätsmedizin Berlin
Germany (DE)
Erasmus University Medical Center (MC)
Netherlands (NL)
Marseille Medical Genetics (MMG)
France (FR)
How to cite
APA:
Yuen, K.C., Vila, G., Bernabeu, I., Buchfelder, M., Freda, P., Henocque, R.,... Brue, T. (2026). Long-term safety and efficacy of pegvisomant monotherapy for acromegaly: final data from the full ACROSTUDY cohort. Pituitary, 29(3). https://doi.org/10.1007/s11102-026-01673-x
MLA:
Yuen, Kevin C.J., et al. "Long-term safety and efficacy of pegvisomant monotherapy for acromegaly: final data from the full ACROSTUDY cohort." Pituitary 29.3 (2026).
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