Does Pegvisomant work?
Yes, in a controlled trial of 112 patients.
The trial was a randomized, double-blind, placebo-controlled study of 112 patients with acromegaly over 12 weeks, in which IGF-1 normalized in a large majority at the highest trial dose. The ACROSTUDY registry in the label followed 2,221 treated patients for a mean of 8.5 years for safety. The trial measured a biochemical endpoint, IGF-1, and not long-term outcomes, but IGF-1 is the accepted marker of disease activity. 12
The trial randomized 112 patients with active acromegaly to placebo or one of three daily doses of pegvisomant for 12 weeks. IGF-1 fell in step with the dose. At the highest trial dose, about 89 percent of patients reached the normal range, against about 10 percent on placebo. Soft-tissue swelling, heavy sweating and fatigue improved along with IGF-1. Ring size went down. 1
The label sums up ACROSTUDY, an observational registry of 2,221 patients treated for a mean of 8.5 years. Liver enzyme rises above five times the upper limit occurred in 1.7% of those with normal tests at the start. On repeat MRI, the pituitary tumor was reported larger in 7.1%, smaller in 17.3% and unchanged in 71.1%. 2
Evidence checklist
- Randomised controlled trial Double-blind placebo-controlled, 12 weeks
- Human study over 100 people 112 acromegaly patients
- More than one research group Trial plus registry data from 20 years
- Clinical outcomes, not surrogate markers IGF-1 normalization, a biochemical marker
- Human safety data beyond case reports Liver enzymes tracked; registries
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More on Pegvisomant
Citations
- Trainer 2000, N Engl J Med: treatment of acromegaly with the growth hormone receptor antagonist pegvisomant doi.org
- Pfizer: Somavert (pegvisomant) prescribing information labeling.pfizer.com
Reference information only, not medical advice. Drawn from the Pegvisomant article, fact-checked against the cited sources. No dosing anywhere on this site.