Optic Pathway Glioma and Endocrine Disorders in Patients With and Without NF1
2Sackler Faculty of Medicine, Tel Aviv University
3Pediatric Hematology-Oncology Department, Schneider Children’s Medical Center of Israel
4Neurologic Unit, Schneider Children’s Medical Center of Israel
Objective:
To assess the prevalence of endocrine dysfunction in children with optic pathway gliomas (OPGs), and to compare outcomes between those with and without neurofibromatosis type 1 (NF1).
Study Design:
A retrospective single center study using data of children diagnosed with OPGs during 1990-2020, followed at a pediatric tertiary center endocrine clinic. Growth and puberty parameters, and occurrence of endocrine dysfunction were evaluated.
Results:
The study included 59 patients (29 males); 36 (61%) with NF1. Isolated optic nerve involvement was higher among patients with than without NF1 (p<0.01). Patients without NF1 were younger at OPG diagnosis (p=0.04); and more often treated with debulking surgery (p<0.01) or chemotherapy (p<0.01), At the last endocrine evaluation, patients without NF1 had comparable height SDS (p=0.45), higher BMI SDS (p=0.02), and a higher rate of endocrine complications (78.3% vs. 41.7%, p=0.006).
Age at the last endocrine evaluation was associated with increased incidence of endocrine disorders (OR=1.42, CI=1.10-1.83, p=0.007). Older age at diagnosis of OPG was associated with a lower risk of developing endocrine disorders (OR=0.54, CI=0.33-0.90, p=0.018). OPG location in the hypothalamus or adjacent structures was associated with a higher risk of developing an endocrine disorder (OR=11.43, CI=1.02-127.82, p=0.048).
Conclusions:
Among patients with OPG, endocrine dysfunction presented more commonly and at a younger age in those without NF1 than with NF1. This may be associated with tumor location and more aggressive treatments. The findings may dictate separate concerns in the follow-up of children with OPG with and without NF1.
