Brazilian multicenter study on pegvisomant treatment in acromegaly

Area of Science:

• Endocrinology
• Pharmacology
• Clinical Medicine

Background:

• Acromegaly is a chronic condition caused by excess growth hormone (GH), leading to significant morbidity.
• Previous therapies for acromegaly include surgery, radiotherapy, somatostatin receptor ligands, and cabergoline, but many patients remain uncontrolled.
• Pegvisomant (PEGV) is a GH receptor antagonist used for acromegaly management.

Purpose of the Study:

• To investigate the real-world therapeutic response and safety of pegvisomant (PEGV) in Brazilian acromegaly patients.
• To identify predictors of treatment response to PEGV in this population.
• To evaluate the impact of PEGV on glycemic control and tumor characteristics.

Main Methods:

• A retrospective multicenter study reviewed data from 109 acromegaly patients treated with PEGV in Brazil.
• Efficacy was defined by achieving normal Insulin-like Growth Factor-I (IGF-I) levels during treatment.
• Predictors of response were analyzed by comparing controlled versus uncontrolled patient groups.

Main Results:

• 74.1% of patients achieved normal IGF-I levels with PEGV treatment.
• Glycemic control improved in 56.6% of patients with pre-diabetes or diabetes.
• Tumor enlargement occurred in 6.5% and liver enzyme elevation in 9.2% of patients; PEGV was discontinued in 6 patients.

Conclusions:

• Pegvisomant (PEGV) demonstrates high efficacy and a favorable safety profile in a real-world setting for acromegaly patients.
• PEGV is an effective treatment option for patients whose acromegaly is not adequately controlled by other therapies.
• Treatment duration and baseline GH and IGF-I levels were identified as predictors of response.