Effectiveness and safety of pegvisomant: a systematic review and meta-analysis of observational longitudinal studies

Acromegaly is a rare disease that often requires drug treatment to achieve control, with pegvisomant being one of the most widely used therapies. In the present paper, we aimed to obtain evidence regarding the effectiveness and safety of pegvisomant by reviewing real-world observational longitudinal studies.

A systematic review was performed with a meta-analysis of event rates (95% confidence interval (CI)) using a random effects model. Sensitivity and subgroup analyses were performed (comprehensive meta-analysis 2.0). The systematic review was performed in accordance to preferred reporting items for systematic reviews and meta-analyses, meta-analysis of observational studies in epidemiology, and Cochrane recommendations (PROSPERO register CRD 42017059880). PubMed, Scopus, Web of Science, and SciELO were used to search for literature. Observational studies in patients using pegvisomant for the treatment of acromegaly were included.

Initially, 552 papers were retrieved from the databases; and 31 articles were included in the qualitative analysis and 14 in the quantitative analysis. Eight primary meta-analyses were performed. The overall rate of patients with disease control was of 60.9% (51.8-69.3%; 95% CI). When considering patients under monotherapy, the control rate was 71.7% (64.0-78.4%; 95% CI). Tumor growth was estimated in 7.3% (4.7-11.1%; 95% CI) and elevation of transaminases in 3.0% (1.7-5.2%; 95% CI).

The real-world data showed that the effectiveness of pegvisomant is not as high as reported in interventional studies. Acromegaly appears to be better controlled when pegvisomant is used as a monotherapy. No serious adverse events were associated with the use of pegvisomant; however, given the high cost of this drug, further studies are required.