AffiliationDepartment of Endocrinology, Christie Hospital, Wilmslow Road, Manchester, M20 4BX, UK.
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AbstractConventional treatments for acromegaly include surgery, radiotherapy, dopamine agonists and somatostatin (SMS) analogues, which effect disease control by lowering circulating growth hormone (GH). Due to variability in tumour characteristics, combinations of these treatment modalities leave a significant number of patients with sub-optimal serum GH and insulin-like growth factor-I (IGF-I) levels, which have been linked to increased morbidity and mortality. The GH receptor antagonist pegvisomant is a genetically engineered analogue of GH that prevents functional dimerisation of the growth hormone receptor (GHR); a process that is critical to GH action at the cellular level. A crucial amino acid substitution at Gly(120) to Arg(120) within the third alpha helix of the antagonist prevents functional GHR dimerisation. Pegvisomant represents a novel treatment for acromegaly as, unlike existing treatment modalities, the effectiveness of pegvisomant is independent of pituitary tumour characteristics. Initial clinical studies in patients with active acromegaly have demonstrated serum IGF-I normalisation in over 90% of patients receiving 20 mg per day, such that, in terms of serum IGF-I normalisation, pegvisomant now represents the most effective medical treatment for acromegaly. Although there are limited long-term data on the use of pegvisomant and questions regarding pituitary tumour growth and altered liver function remain, this therapy offers the prospect of serum IGF-I normalisation in the vast majority of patients with active acromegaly.
CitationThe place of pegvisomant in the management of acromegaly. 2001, 10 (9):1725-35 Expert Opin Investig Drugs
JournalExpert Opinion on Investigational Drugs
- Pegvisomant: an advance in clinical efficacy in acromegaly.
- Authors: Stewart PM
- Issue date: 2003 Apr
- Pegvisomant in the treatment of acromegaly.
- Authors: Parkinson C, Scarlett JA, Trainer PJ
- Issue date: 2003 Sep 26
- Experience with pegvisomant in the treatment of acromegaly.
- Authors: Drake WM
- Issue date: 2001 Jun
- The place of pegvisomant in the acromegaly treatment algorithm.
- Authors: Paisley AN, Trainer PJ, Drake WM
- Issue date: 2004 Jun
- Pegvisomant: a growth hormone receptor antagonist used in the treatment of acromegaly.
- Authors: Tritos NA, Biller BM
- Issue date: 2017 Feb