Acromegaly: pegvisomant disappoints in meta-analysis of real-world data

  • Leonart LP & al.
  • Endocrine
  • 25 Aug 2018

  • curated by Brian Richardson, PhD
  • Clinical Essentials
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Takeaway

  • A systematic review and meta-analysis suggest that pegvisomant treatment is associated with a 61% control rate in patients with acromegaly.

Why this matters

  • This meta-analysis focuses on real-world data of pegvisomant (a genetically modified human growth hormone analog) effectiveness and finds at least 25% lower rates than in interventional studies.

Key results

  • The overall disease control rate was 60.9% (95% CI, 51.8%-69.3%) in pegvisomant-treated patients.
  • The disease control rate was 71.7% (95% CI, 64.0%-78.4%) in patients treated with pegvisomant monotherapy.
  • The overall tumor growth rate was 7.3% (95% CI, 4.7%-11.1%).
    • 7.2% (95% CI, 4.8%-10.7%) in monotherapy patients.
  • Elevated transaminase levels were estimated to occur in 3.0% (95% CI, 1.7%-5.2%) of patients.

Study design

  • 31 real-world, observational, longitudinal articles including patients with acromegaly treated with pegvisomant were included in qualitative analysis and 14 were included in quantitative analysis, with 8 primary meta-analyses performed.
  • Funding: The Brazilian National Council of Technological and Scientific Development and the Coordination for the Improvement of Higher Education Personnel provided scholarships.

Limitations

  • Only observational studies were included.

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