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. 2008 Aug;93(8):2953-6.
doi: 10.1210/jc.2007-2283. Epub 2008 May 20.

Treatment of pituitary gigantism with the growth hormone receptor antagonist pegvisomant

Naila Goldenberg  1 , Michael S Racine, Pamela Thomas, Bernard Degnan, William Chandler, Ariel Barkan
Affiliations

Treatment of pituitary gigantism with the growth hormone receptor antagonist pegvisomant

Naila Goldenberg et al. J Clin Endocrinol Metab. 2008 Aug.

Abstract

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.

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Figures

Figure 1
Figure 1
Clinical course of three patients with gigantism. Upper row, Actual growth curves. Middle row, Annualized GVs. Lower row, Plasma IGF-I concentrations (shaded areas represent normal ranges). Left column, Patient 1. Center column, Patient 2. Right column, Patient 3. Sx, Surgery; DA, dopamine agonist; LAR, octreotide LAR; Pg, pegvisomant; SP, spontaneous puberty; T, exogenous testosterone. Arrows mark the time of surgery.
Figure 2
Figure 2
Expansion of pituitary tumor size in patient 2. Left panel, Coronal view of the sellar area after about 2 yr of combined octreotide LAR (20 mg every month) and pegvisomant (20 mg every day) therapy. Right panel, Same view after another year of therapy with octreotide LAR and pegvisomant in the same doses.
See this image and copyright information in PMC

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