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Growth Hormone Related Diseases and Therapy pp 339–357Cite as

GHR Antagonist: Efficacy and Safety

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Part of the book series: Contemporary Endocrinology ((COE))

Abstract

Pegvisomant is a GH receptor antagonist (GHR) licensed for the treatment of acromegaly. The story of the development of pegvisomant began with the recognition of the therapeutic implications of GH gene transgenic mice having a dwarf, rather than giant, phenotype and culminated in studies demonstrating its ability to normalise IGF-I in up to 97% of patients. The defining studies used pegvisomant as a daily monotherapy but more recent data suggest pegvisomant can be as effective when administered as a weekly preparation or in combination with somatostatin analogues or dopamine agonists. It is well tolerated and available data do not provide any evidence of potentiation of tumour growth. Approximately 3% of patients experience elevation in liver transaminases, typically within weeks of commencing treatment. The aetiology remains uncertain and available data suggest that it resolves on discontinuation of the drug. Important long term safety and efficacy data will continue to be provided by the ACROSTUDY registry that is open to all patients receiving pegvisomant.

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Authors and Affiliations

  1. Department of Endocrinology, Christie Hospital, Manchester, UK

    Claire E. Higham

  2. Department of Endocrinology, Christie Hospital, Manchester, M20 4BX, UK

    Peter J. Trainer

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    Ken Ho

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Higham, C.E., Trainer, P.J. (2011). GHR Antagonist: Efficacy and Safety. In: Ho, K. (eds) Growth Hormone Related Diseases and Therapy. Contemporary Endocrinology. Humana Press. https://doi.org/10.1007/978-1-60761-317-6_17

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