OBJECTIVES: Acromegaly is an endocrine rare disease that often requires drug treatment. Pegvisomant is the most effective medical therapy to treat this disease, and it is on the market for more than 10 years. Therefore, we aimed to gather evidence regarding the effectiveness and safety of pegvisomant by reviewing real-world observational longitudinal studies.

METHODS: A systematic review was performed with a meta-analysis of event rates (95% confidence interval – CI) using a fixed effects model. Sensitivity and subgroup analyses were performed (Comprehensive Meta-analysis 2.0). The systematic review was conducted in accordance to PRISMA, MOOSE, and Cochrane recommendations (PROSPERO CRD42017060756). PubMed, Scopus, Web of Science, and SciELO were used to search for literature. Longitudinal observational studies in patients using pegvisomant for the treatment of acromegaly were included.

RESULTS: Initially, 552 papers were retrieved from the databases; 31 were included in the qualitative analysis and 12 in the quantitative analysis. Two primary meta-analyses on pegvisomant effectiveness were performed. The overall rate of patients with disease control (normal IGF-1 level for sex and age) was 56.2% (52.8–59.6%; 95% CI). When considering patients under monotherapy, the control rate was 71.6% (64.5–77.8%; 95% CI). Subgroup analyses considering data over 1, 2, 3, 4, and 5 years of treatment were conducted and similar results were observed. Sensitivity analyses did not affect the results. Additional analyses showed a good safety profile.

CONCLUSIONS: The meta-analysis of real-world data showed that the effectiveness of pegvisomant is not as high as reported in interventional studies. Our results demonstrated that the performance of pegvisomant is homogeneous over time, which is important given the chronicity of the disease. Acromegaly appears to be better controlled when pegvisomant is used as a monotherapy. Pegvisomant is effective in treating acromegaly; however, the high costs of this drug impairs a greater use, above all as monotherapy.