Reversible Growth Hormone Excess in Two Girls with Neurofibromatosis Type 1 and Optic Pathway Glioma.
BACKGROUND
A total of 12 children with neurofibromatosis type 1 (NF-1) with optic pathway glioma (OPG) and growth hormone (GH) excess are reported to date, but no data exist on the long-term outcome. We describe 2 girls with NF-1 with OPG and GH excess treated with somatostatin analogue (SSa) who maintained a normal GH axis after stopping SSa therapy.


METHODS
The diagnosis of GH excess was established from auxological data, persistently high levels of insulin-like growth factor 1 (IGF-1) and a lack of GH suppression during an oral glucose tolerance test.


RESULTS
Both patients were started on SSa treatment. During treatment, growth deceleration and normal IGF-1 levels were documented. The first case stopped treatment following the development of SSa side effects. The second case interrupted SSa when, closed to her final height, a normal IGF-1 level was documented. While off treatment, both cases maintained normal IGF-1 levels and appropriate growth velocity for their age and development, with normal GH secretion on biochemical testing. Both cases received treatment for central precocious puberty.


CONCLUSIONS
GH excess in NF-1 children with OPG can be reversed and only short-term SSa therapy may be required. The aetiology remains undetermined, but the course suggests a hypothalamic dysfunction.
Pediatric Department, Royal Marsden NHS Foundation Trust, London, UK.
Patrizia
P
Bruzzi
Ilaria
I
Sani
Assunta
A
Albanese
D000172:Acromegaly / Q000150:complications / Q000188:drug therapy*; D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D005910:Glioma / Q000150:complications* / Q000473:pathology; D006801:Humans; D009456:Neurofibromatosis 1 / Q000150:complications* / Q000473:pathology; D015282:Octreotide / Q000627:therapeutic use*; D019574:Optic Nerve Neoplasms / Q000150:complications* / Q000473:pathology; D016896:Treatment Outcome
D015282:Octreotide