Pegvisomant in acromegaly: a multicenter real-life study in Argentina

Arch Endocrinol Metab. 2019 Aug 22;63(4):320-327. doi: 10.20945/2359-3997000000160.

Abstract

Objective: To describe the long term safety and efficacy of pegvisomant (PEGV), and the predictors of treatment response in patients with acromegaly in the real life setting.

Subjects and methods: We retrospectively reviewed the clinical, hormonal and radiological data of acromegalic patients treated with PEGV in 17 Argentine centers.

Results: Seventy-five patients (age range 22-77, 51 females) with acromegaly have been treated with PEGV for up to 118 months (median 27 months). Before PEGV, 97.3% of patients had been treated with medical therapy, surgery and/or radiotherapy, two patients had no previous treatment. At that time, all patients had an IGF-1 above the upper normal limit (ULN) (mean 2.4 x ULN ± 0.98, range 1.25-7). At diagnosis of acromegaly 84% presented macroadenomas, prior to PEGV only 23,5% of patients remained with tumor remnant > 1 cm, the remaining showed normal or less than 1 cm images. Disease control (IGF-1 ≤ 1.2 x ULN) was achieved in 62.9% of patients with a mean dose of 11.8 mg/day. Thirty-four patients (45%) received PEGV monotherapy, while 41 (55%) received combined therapy with either somatostatin analogues and/or cabergoline. Adverse events related to PEGV were: local injection site reaction in 5.3%, elevated liver enzymes in 9.3%, and tumor size growth in 9.8%. Pre-PEGV IGF-I level was the only predictor of treatment response: 2.1 x ULN vs 2.8 x ULN in controlled and uncontrolled patients respectively (p < 0.001).

Conclusion: this long term experience indicates PEGV treatment was highly effective and safe in our series of Argentine patients with acromegaly refractory to standard therapies. Arch Endocrinol Metab. 2019;63(4):320-7.

Publication types

  • Multicenter Study
  • Observational Study

MeSH terms

  • Acromegaly / drug therapy*
  • Adult
  • Aged
  • Argentina
  • Cabergoline / administration & dosage
  • Cabergoline / therapeutic use*
  • Dopamine Agonists / administration & dosage
  • Dopamine Agonists / therapeutic use*
  • Drug Therapy, Combination
  • Female
  • Follow-Up Studies
  • Human Growth Hormone / administration & dosage
  • Human Growth Hormone / analogs & derivatives*
  • Human Growth Hormone / therapeutic use
  • Humans
  • Insulin-Like Growth Factor I / analysis
  • Male
  • Middle Aged
  • Predictive Value of Tests
  • Retrospective Studies
  • Somatostatin / administration & dosage
  • Somatostatin / analogs & derivatives*
  • Somatostatin / therapeutic use
  • Treatment Outcome
  • Young Adult

Substances

  • Dopamine Agonists
  • IGF1 protein, human
  • Human Growth Hormone
  • Somatostatin
  • Insulin-Like Growth Factor I
  • Cabergoline
  • pegvisomant