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

Abstract

OBJECTIVE:

To report the final long-term safety and efficacy analyses of patients 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 acromegaly, 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) and 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 occurred 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 combination therapy in patients with an aggressive course/uncontrolled/active acromegaly requiring long-term medical therapy for control.

• Authors: 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
• Journal: European journal of endocrinology (Eur J Endocrinol) Vol. 185 Issue 4 Pg. 525-538 (Aug 27 2021) ISSN: 1479-683X [Electronic] England
• PMID: 34342594 (Publication Type: Historical Article, Journal Article, Multicenter Study)

Chemical References

• Human Growth Hormone
• pegvisomant

Topics

• Acromegaly (drug therapy, epidemiology)
• Adenoma (drug therapy, epidemiology)
• Adolescent
• Adult
• Aged
• Aged, 80 and over
• Child
• Child, Preschool
• Cohort Studies
• Europe (epidemiology)
• Female
• Follow-Up Studies
• Growth Hormone-Secreting Pituitary Adenoma (drug therapy, epidemiology)
• History, 21st Century
• Human Growth Hormone (analogs & derivatives, therapeutic use)
• Humans
• Infant
• Male
• Middle Aged
• Retrospective Studies
• Treatment Outcome
• United States (epidemiology)
• Young Adult