Journal of Clinical Endocrinology & Metabolism, 91(8), 2960–2966 (2006), DOI:10.1210/jc.2005-2661

Pegvisomant for the Treatment of it gsp-Mediated Growth Hormone Excess in Patients with McCune-Albright Syndrome

S. O. Akintoye, M. H. Kelly, B. Brillante, N. Cherman, S. Turner, J. A. Butman, P. G. Robey, M. T. Collins

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, Gsα (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.

back


© 2021 ALL RIGHTS RESERVED
The abstract is a scientific citation from the corresponding source. The copyright of the publisher is not affected by this citation.

Ihr Browser versucht gerade eine Seite aus dem sogenannten Internet auszudrucken. Das Internet ist ein weltweites Netzwerk von Computern, das den Menschen ganz neue Möglichkeiten der Kommunikation bietet.

Da Politiker im Regelfall von neuen Dingen nichts verstehen, halten wir es für notwendig, sie davor zu schützen. Dies ist im beidseitigen Interesse, da unnötige Angstzustände bei Ihnen verhindert werden, ebenso wie es uns vor profilierungs- und machtsüchtigen Politikern schützt.

Sollten Sie der Meinung sein, dass Sie diese Internetseite dennoch sehen sollten, so können Sie jederzeit durch normalen Gebrauch eines Internetbrowsers darauf zugreifen. Dazu sind aber minimale Computerkenntnisse erforderlich. Sollten Sie diese nicht haben, vergessen Sie einfach dieses Internet und lassen uns in Ruhe.

Die Umgehung dieser Ausdrucksperre ist nach §95a UrhG verboten.

Mehr Informationen unter www.politiker-stopp.de.