Treatment of pituitary gigantism with the growth hormone receptor antagonist pegvisomant.
BACKGROUND
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.


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


METHODS
The study was conducted at a university hospital.


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


METHODS
The intervention was administration of pegvisomant.


METHODS
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.
Goldenberg
Naila
N
University of Michigan Medical Center, Division of MEND, Department of Internal Medicine, Ann Arbor, Michigan 48109, USA.
Racine
Michael S
MS
Thomas
Pamela
P
Degnan
Bernard
B
Chandler
William
W
Barkan
Ariel
A
D000293:Adolescent; D000365:Age Determination by Skeleton; D002648:Child; D005260:Female; D005877:Gigantism / Q000097:blood / Q000188:drug therapy* / Q000503:physiopathology; D006128:Growth; D019382:Human Growth Hormone / Q000031:analogs & derivatives* / Q000627:therapeutic use; D006801:Humans; D007334:Insulin-Like Growth Factor I / Q000032:analysis; D008297:Male; D010911:Pituitary Neoplasms / Q000473:pathology; D011986:Receptors, Somatotropin / Q000037:antagonists & inhibitors*
D011986:Receptors, Somatotropin; D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I; C406545:pegvisomant