More than a decade of real-world experience of pegvisomant for acromegaly: ACROSTUDY

Maria Fleseriu, Dagmar Führer-Sakel, Aart J. van der Lely, Laura De Marinis, Thierry Brue, Joli van der Lans-Bussemaker, Judith Hey-Hadavi, Cecilia Camacho-Hubner, Michael P. Wajnrajch, Srinivas Rao Valluri, Andrew Anthony Palladino, Roy Gomez, Roberto Salvatori

Research output: Contribution to journalArticlepeer-review

10 Scopus citations

Abstract

Objective: To report the final long-term safety and efficacy analyses of pati ents with acromegaly treated with pegvisomant from the ACROSTUDY. Design: Global (15 countries), multicentre, non-interventional study (2004-2017). Methods: The complete ACROSTUDY cohort comprised patients with acromegal y, who were being treated with pegvisomant (PEGV) prior to the study or at enrolment. The main endpoints were long-term safety (comorbidities, adverse events (AEs), pituitary tumour volumes, liver tests) an d efficacy (IGF1 changes). Results: Patients (n = 2221) were treated with PEGV for a median of 9.3 years (range, 0-20.8 years) and followed up for a median of 7.4 years (range, 0-13.9 years). Before PEGV, 96.3% had received other acromegaly treatments (surgery/ radiotherapy/medications). Before PEGV treatment, 87.2% of patients reported comorbidities. During ACROSTUDY, 5567 AEs were reported in 56.5% of patients and of these 613 were considered treatment-related (in 16.5% of patients) and led to drug withdrawal in 1.3%. Pituitary imaging showed a tumour size increase in 7.1% of patients; the majority (71.1%) reported no changes. Abnormal AST or ALT liver tests oc curred in 3.2% of patients. IGF1 normalization rate improved over time, increasing from 11.4% at PEGV start to 53.7 % at year 1, and reaching 75.4% at year 10 with the use of ≥30 mg PEGV/day in an increasing proportion of patients. Conclusion: This comprehensive review of the complete cohort in ACROSTUDY confirmed the overall favourable benefit-to-risk profile and high efficacy of PEGV as mono- and comb ination therapy in patients with an aggressive course/uncontrolled/active acromegaly requiring long-term medical therapy for control.

Original languageEnglish (US)
Pages (from-to)525-538
Number of pages14
JournalEuropean journal of endocrinology
Volume185
Issue number4
DOIs
StatePublished - Oct 2021

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Endocrinology

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