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Pegvisomant: a growth hormone receptor antagonist used in the treatment of acromegaly

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Abstract

Purpose

To review published data on pegvisomant and its therapeutic role in acromegaly.

Methods

Electronic searches of the published literature were conducted using the keywords: acromegaly, growth hormone (GH) receptor (antagonist), pegvisomant, therapy. Relevant articles (n = 141) were retrieved and considered for inclusion in this manuscript.

Results

Pegvisomant is a genetically engineered, recombinant growth hormone receptor antagonist, which is effective in normalizing serum insulin-like growth factor 1 (IGF-1) levels in the majority of patients with acromegaly and ameliorating symptoms and signs associated with GH excess. Pegvisomant does not have direct antiproliferative effects on the underlying somatotroph pituitary adenoma, which is the etiology of GH excess in the vast majority of patients with acromegaly. Therefore, patients receiving pegvisomant monotherapy require regular pituitary imaging in order to monitor for possible increase in tumor size. Adverse events in patients on pegvisomant therapy include skin rashes, lipohypertrophy at injection sites, and idiosyncratic liver toxicity (generally asymptomatic transaminitis that is reversible upon drug discontinuation), thus necessitating regular patient monitoring.

Conclusions

Pegvisomant is an effective therapeutic agent in patients with acromegaly who are not in remission after undergoing pituitary surgery. It mitigates excess GH action, as demonstrated by IGF-1 normalization, but has no direct effects on pituitary tumors causing acromegaly. Regular surveillance for possible tumor growth and adverse effects (hepatotoxicity, skin manifestations) is warranted.

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Authors and Affiliations

  1. Neuroendocrine Unit, Massachusetts General Hospital, Zero Emerson Place # 112, Boston, MA, 02114, USA

    Nicholas A. Tritos & Beverly M. K. Biller

  2. Harvard Medical School, Boston, MA, USA

    Nicholas A. Tritos & Beverly M. K. Biller

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Correspondence to Nicholas A. Tritos.

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NAT has received institution-directed research support from Ipsen, Novartis, Novo Nordisk and Pfizer and has consulted for Ipsen. BMKB has received institution-directed research support from Novartis and has consulted for Novartis, Ipsen and Pfizer.

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Tritos, N.A., Biller, B.M.K. Pegvisomant: a growth hormone receptor antagonist used in the treatment of acromegaly. Pituitary 20, 129–135 (2017). https://doi.org/10.1007/s11102-016-0753-y

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