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Long-term effects of pegvisomant in patients with acromegaly
1Department of Internal Medicine, Division of Metabolism, Endocrinology and Diabetes, University of Michigan Medical Center and the Veterans' Affairs Medical Center, Ann Arbor, MI, USA.
Abstract:
Despite improved surgical and radiotherapy techniques and the development of long-acting somatostatin analogs, some patients with acromegaly cannot attain biochemical remission. As a consequence they continue to endure debilitating symptoms and mortality remains high. Pegvisomant, a recombinant growth-hormone-receptor antagonist, suppresses production of insulin-like growth factor I. Since the introduction of this drug several years ago, long-term studies involving hundreds of patients have established efficacy of more than 85%. Raised transaminase concentrations have, however, been reported as a side effect of therapy, albeit an infrequent one. In addition, increases in tumor volume have been reported in several cases. In this Review we present the long-term data that have been gathered on pegvisomant therapy, discuss the related risks and benefits, and frame a potential therapeutic approach.
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