Pegvisomant treatment in a 4-year-old girl with neurofibromatosis type 1.
OBJECTIVE
Growth hormone (GH) excess in childhood is a rare disorder. Current treatment options such as somatostatin analogues, pituitary surgery or irradiation can have serious side effects. Recently, a GH receptor antagonist, pegvisomant, was introduced for the treatment of adults with acromegaly. We wanted to investigate whether pegvisomant was effective in a child with octreotide-resistant GH excess.


METHODS
A 4-year-old girl with neurofibromatosis type 1 and GH excess associated with optic glioma received pegvisomant injections (10 mg subcutaneously) with increasing intervals from daily to every 4th day.


RESULTS
IGF-I and IGFBP-3 decreased from +6.9 and 4.6 standard deviation scores (SDS), respectively, to within normal range. Height velocity dropped from 12.4 SDS to mean -0.7 SDS (range: -5.0 to 5.0) and height SDS decreased from +1.3 to +0.6 (target height: +0.2). Random non-fasting serum GH values were mean 5.0 mlU/l (range: 1.6-9.5). There was no change in fasting blood glucose (4.6-4.7 mmol/l) or glycosylated haemoglobin (5.5%) and no subjective or biochemical side effects. Repeated tests of thyroid, adrenal and gonadal function showed no alterations during the treatment period. Intracranial tumours remained unchanged in size and visual impairment did not deteriorate.


CONCLUSIONS
Pegvisomant normalized IGF-I and IGFBP-3 levels. Growth velocity was normalized after initial catch-down growth, and it remains to be seen whether this result can be maintained during long-term treatment.
Main
Katharina M
KM
University Department of Growth and Reproduction, Rigshospitalet, Copenhagen, Denmark. katharina.main@rh.hosp.dk
Sehested
Astrid
A
Feldt-Rasmussen
Ulla
U
D000970:Antineoplastic Agents / Q000627:therapeutic use; D001827:Body Height / Q000187:drug effects; D002675:Child, Preschool; D005260:Female; D019382:Human Growth Hormone / Q000031:analogs & derivatives* / Q000627:therapeutic use; D006801:Humans; D018972:Insulin-Like Growth Factor Binding Protein 3 / Q000097:blood; D007334:Insulin-Like Growth Factor I / Q000378:metabolism; D009456:Neurofibromatosis 1 / Q000150:complications / Q000188:drug therapy*; D020339:Optic Nerve Glioma / Q000150:complications / Q000188:drug therapy*
D000970:Antineoplastic Agents; D018972:Insulin-Like Growth Factor Binding Protein 3; D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I; C406545:pegvisomant