Growth hormone excess in children with neurofibromatosis and optic pathway glioma, an underdiagnosed condition: experience with long-acting treatment.
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BACKGROUND: Growth hormone excess (GHE) in children with neurofibromatosis Type 1 (NF1) has been reported in some cases. The prevalence of GHE in NF1 has not been described and treatment with long-acting somatostatin analogues (SSAs) has not been well characterized. OBJECTIVE: To describe in children with NF1/optic pathway glioma (OPG) the prevalence of GHE and response to SSA. RESULTS: From 379 children with NF1, 80 were diagnosed of OPG (21%). In a prospective follow-up, 8.7% were identified as having GHE; all were prepubertal, with a mean age of 4.4 ± 1.9 years. The mean height was +0.86 ± 0.76 SD above the mid-parental height and growth velocity +4.07 SD, mean insulin-like growth factor 1 (IGF-1) > 1 SD (457.8 ± 151.3 ng/mL). In 4 patients, the GHE was observed during tumour progression. The first 2 patients were initially treated with short-acting SSAs (1.5 μg/kg/day). After confirming tolerability, it was replaced by long-acting SSAs (10 mg/28 days). Four were initially treated with (10 mg/28 d). 6/7 patients showed a normalization of IGF-1 and growth velocity. Treatment could be withdrawn in all patients after 19.9 ± 4.6 months. The patients remained stable for 48 months (24-60). Except for mild diarrhoea, no other adverse events were observed. CONCLUSIONS: 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.