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Pegvisomant for the treatment of gsp-mediated growth hormone excess in patients with McCune-Albright syndrome.

Authors: Akintoye, Sunday O  Kelly, Marilyn H  Brillante, Beth  Cherman, Natasha  Turner, Sarah  Butman, John A  Robey, Pamela G  Collins, Michael T 
Citation: Akintoye SO, etal., J Clin Endocrinol Metab. 2006 Aug;91(8):2960-6. Epub 2006 May 23.
Pubmed: (View Article at PubMed) PMID:16720661
DOI: Full-text: DOI:10.1210/jc.2005-2661

CONTEXT: GH excess affects approximately 20% of the patients with McCune-Albright syndrome (MAS). MAS is caused by sporadic, postzygotic, activating mutations in the GNAS gene, which codes for the cAMP-regulating protein, G(s)alpha (gsp oncogene). These same mutations are found in approximately one third of the sporadic cases of acromegaly.
OBJECTIVE: We examined efficacy of the GH receptor antagonist, pegvisomant, in controlling gsp oncogene-mediated GH excess and skeletal disease (fibrous dysplasia of bone) associated with MAS.
SETTING AND PATIENTS: Five MAS patients with GH excess were treated with 20 mg/d sc injection of pegvisomant for 12 wk in a randomized, double-blind, placebo-controlled crossover study at the National Institutes of Health.
MAIN OUTCOME MEASURES: The primary measure of efficacy was normalization of IGF-I. Secondary outcome measures were reduction in serum IGF binding protein-3 (IGFBP-3), improvement of fatigue and sweating, and reduction in markers of bone metabolism and bone pain.
RESULTS: Combined mean changes in serum IGF-I at 6 and 12 wk were -236.4 ng/ml (53%, P < 0.005) and -329.8 ng/ml (62%, P < 0.001), respectively. IGFBP-3 decreased by 0.8 mg/liter (24%, P < 0.01) and 2.9 mg/liter (37%, P < 0.005), respectively. There were no significant changes in signs and symptoms of acromegaly or markers of bone metabolism and bone pain, nor was there a significant change in pituitary size. Retrospective comparison of the degree of control achieved with pegvisomant vs. other medications (long-acting octreotide +/- dopamine agonist) in the same group showed that the two regimens were similarly effective.
CONCLUSIONS: Pegvisomant effectively reduced IGF-I and IGFBP-3 levels in gsp-mediated GH excess but had no effect on fibrous dysplasia.


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CRRD Object Information
CRRD ID: 12743609
Created: 2017-02-09
Species: All species
Last Modified: 2017-02-09
Status: ACTIVE


RGD is funded by grant HL64541 from the National Heart, Lung, and Blood Institute on behalf of the NIH.