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Published on: January 14, 2014
Natural history and outcome of optic pathway gliomas in children
Gary Nicolin1, Patricia Parkin, Donald Mabbott
1Pediatric Brain Tumor Program, Division of Hematology/Oncology, Hospital for Sick Children, Toronto, Ontario, Canada.
Insights
Management of optic pathway gliomas (OPGs) is complex, especially with neurofibromatosis type 1 (NF1). This study found NF1 significantly influences tumor behavior, impacting treatment strategies and outcomes in children.
Area of Science:
- Pediatric Oncology
- Neuro-oncology
- Ophthalmology
Background:
- Optic pathway gliomas (OPGs) present complex management challenges due to variable disease progression and association with neurofibromatosis type 1 (NF1).
- Defining tumor progression and response to therapy in OPGs remains difficult, complicating treatment decisions.
Purpose of the Study:
- To retrospectively analyze the management and outcomes of pediatric patients with optic pathway gliomas.
- To investigate the influence of neurofibromatosis type 1 (NF1) on the behavior and treatment response of OPGs.
Main Methods:
- Retrospective review of pediatric patients diagnosed with OPGs between 1990 and 2004 at a single institution.
- Analysis of treatment modalities, including observation, chemotherapy, surgery, and radiotherapy, and their impact on progression-free survival (PFS).
Main Results:
- Of 133 children, 59% had NF1. Initial observation was common, with 23% of observed patients eventually requiring treatment.
- Chemotherapy alone was the most frequent initial treatment. Overall survival at 5 and 10 years was high for treated patients (97.6% and 94.6%).
- Progression-free survival was 48% for treated patients, with no significant difference in PFS based on chemotherapy, debulking, or combined approaches. NF1 status did not significantly alter PFS.
Conclusions:
- Optic pathway gliomas are complex tumors, with NF1 being a critical factor influencing tumor behavior.
- Management strategies for OPGs require careful consideration of individual tumor characteristics and the presence of NF1.
Background:
The optimal management of optic pathway gliomas (OPGs) is complicated by their variable natural history, the association with neurofibromatosis type 1 (NF1) and difficulties in defining progression and response to treatment.
Methods:
This study is a retrospective review of all children presenting to a single institution with an OPG between 1990 and 2004.
Results:
Of the 133 children included, 78 (59%) had NF1; 87 (71 NF1) were observed initially, of whom 23 (11 NF1) subsequently required treatment. Forty-six patients received immediate treatment. Initial treatment, without or with an observation period, comprised chemotherapy alone (32, 11 NF1); debulking + chemotherapy (15, 4 NF1); gross total resection (6); radiotherapy (2); debulking + radiotherapy (3); and debulking only (12, 3 NF1). Overall, 16 patients were irradiated during the study period. Four children died (overall survival at 5 and 10 years was 97.6% and 94.6% for those who required treatment). Progression-free survival (PFS) for the 69 patients who needed treatment was 48%. There was no difference in PFS between chemotherapy versus chemotherapy + debulking or debulking alone. PFS for the NF1 patients who required treatment was similar to that of non-NF1 patients. Mean follow-up time was 9.0 (range 0.6-18.0, median 8.6) years.
Conclusions:
The study confirms the complexity of OPGs and that NF1 is a major determinant of the resultant behavior of the tumor.
