Growth hormone excess in children with neurofibromatosis and optic pathway glioma, an underdiagnosed condition: experience with long-acting treatment
Authors' conclusion
We should consider the risk GHE in patients with NF1-OPG, as this may be a cause for concern indicating tumour progression. Treatment with long-acting SSAs was effective and safe. After treatment, the patients' growth and analytical parameters remained within normal range, confirming the reversibility of GHE.
Study context
- Population
- Children with NF1 and optic pathway glioma (OPG)80 with OPG
- Participants
- 379 children
- Outcome
- Prevalence of GH excess and response to SSA
Methodological detailsprospective follow-up
- Prospective follow-up
Main finding
As reported in the abstract
GH excess prevalence in NF1-OPG
OPG among children with NF1
80
21%
GH excess identified in prospective follow-up
8.7%
Normalization of IGF-1 and growth velocity with SSA
6/7 patients
Observational data: associations, not effects.
Further findings
Characteristics at GH excess diagnosis
- All prepubertal; mean age
- 4.4 ± 1.9 years
- Mean height above mid-parental height
- +0.86 ± 0.76 SD
- Growth velocity
- +4.07 SD
- Mean IGF-1
- 457.8 ± 151.3 ng/mL
- GH excess observed during tumour progression
- 4 patients
Treatment course
- First 2 patients: short-acting SSAs then long-acting SSAs (10 mg/28 days)
- 1.5 μg/kg/day
- Initially treated with long-acting SSA (10 mg/28 d)
- 4
- Treatment withdrawn in all patients after (months)
- 19.9 ± 4.6
- Remained stable after withdrawal (months)
- 48 (24-60)
Safety
- Except for mild diarrhoea, no other adverse events were observed
Original publication
Growth hormone excess in children with neurofibromatosis and optic pathway glioma, an underdiagnosed condition: experience with long-acting treatment. Eur J Endocrinol. Published 4 June 2025. doi:10.
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