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Treatment of Pituitary Gigantism with the Growth Hormone Receptor Antagonist Pegvisomant

Division of Metabolism, Endocrinology, and Diabetes, Department of Internal Medicine (N.G., M.S.R., W.C., A.B.) and the Department of Neurosurgery (W.C., A.B.), University of Michigan Medical Center, Ann Arbor, Michigan 48109; Department of Pediatric Endocrinology (P.T.), Lutheran Children’s Hospital, Fort Wayne, Indiana 46804; and Department of Pediatric Endocrinology (B.D.), St. John’s Hospital, Detroit, Michigan 48236

Address all correspondence and requests for reprints to: Ariel L. Barkan, M.D., University of Michigan Medical Center, Division of MEND, 1500 East Medical Center Drive, 3920 Taubman Center, SPC 5354, Ann Arbor, Michigan 48109. E-mail: abarkan{at}umich.edu.

Context: Treatment of pituitary gigantism is complex and the results are usually unsatisfactory.

Objective: The objective of the study was to describe the results of therapy of three children with pituitary gigantism by a GH receptor antagonist, pegvisomant.

Design: This was a descriptive case series of up to 3.5 yr duration.

Setting: The study was conducted at a university hospital.

Patients: Patients included three children (one female, two males) with pituitary gigantism whose GH hypersecretion was incompletely controlled by surgery, somatostatin analog, and dopamine agonist.

Intervention: The intervention was administration of pegvisomant.

Main Outcome Measures: Plasma IGF-I and growth velocity were measured.

Results: In all three children, pegvisomant rapidly decreased plasma IGF-I concentrations. Growth velocity declined to subnormal or normal values. Statural growth fell into lower growth percentiles and acromegalic features resolved. Pituitary tumor size did not change in two children but increased in one boy despite concomitant therapy with a somatostatin analog.

Conclusions: Pegvisomant may be an effective modality for the therapy of pituitary gigantism in children. Titration of the dose is necessary for optimal efficacy, and regular surveillance of tumor size is mandatory.