What question did this study set out to answer?

This research aims to evaluate the long-term visual and endocrine outcomes in children diagnosed with optic pathway gliomas.

June 26, 2026Open Access

ID #349 Beyond tumor control: late effects in pediatric optic pathway glioma in an Low-middle income setting

Key Points

This research aims to evaluate the long-term visual and endocrine outcomes in children diagnosed with optic pathway gliomas.
Included children ≤18 years with optic pathway gliomas diagnosed from January 2011 to December 2022.
Initial treatment was chemotherapy (vincristine–carboplatin, vinblastine, trametinib), with radiotherapy delayed until necessary.
Followed progression-free survival, radiation-free survival, and overall survival using Kaplan–Meier methods.
5-year progression-free survival was 61.2%, radiation-free survival was 76.2%, and overall survival was 87.7%.
At follow-up, 57% experienced poor vision, and 45.8% had endocrine dysfunction.
Irradiated patients showed higher endocrine morbidity (51%) than non-irradiated (22%), p<0.001, without vision improvement.

Abstract

Abstract Background Optic pathway/hypothalamic gliomas (OPHG) comprise approximately 15% of low-grade gliomas. Despite favorable overall survival, these tumors are associated with significant visual and endocrine morbidity. Surgical resection is often limited by location, and radiotherapy (RT) carries substantial long-term toxicity. Methodology Children ≤18 years diagnosed with OPHG between January 2011 and December 2022 were included. Chemotherapy was used as the initial treatment strategy, with vincristine–carboplatin as first line,vinblastine monotherapy in neurofibromatosis (NF)-associated tumors and trametinib in recent years. RT was avoided or delayed and generally administered after failure of two systemic therapy lines or symptomatic progression. Baseline and serial visual and endocrine assessments were performed. Progression-free survival (PFS), radiation-free survival (RaFS), and overall survival (OS) were estimated using Kaplan–Meier methods. Results A total of 142 children were included; median diagnosis age 77 months, 73 (51.4%) male. Tumor location was hypothalamic in 122 (87.1%), optic nerve plus chiasma in 13 (9.3%), and optic nerve alone in 5 (3.6%). Presenting features included visual symptoms (78.2%), raised intracranial pressure (40.8%), diencephalic syndrome (7%), precocious puberty (11.3%), seizures (9.2%), and hemiparesis (4.2%). Thirty-four patients (24%) had NF. Among those tested, BRAF alterations included BRAFV600E mutation (0.5%) and fusion (40%). Three patients were observed; 139 received first-line therapy, 48 second-line, 10 third-line, and 3 fourth-line treatment. Overall, 46 (32.4%) received RT. At a median follow-up of 68months, 5-year PFS was 61.2±4.7% RaFS76.2±4%and OS 87.7±2.5%. At last follow-up, 3.5% were blind, 57% had poor vision, and 45.8% had endocrine dysfunction. Irradiated children had a higher endocrine morbidity (51% vs 22%, p 0.001), but no improvement in vision. Conclusions Although survival outcomes are favorable in OPHG, survivors experience a substantial burden of long-term effects related to tumor location and treatment. Early diagnosis, multidisciplinary management, and long-term rehabilitation are essential to optimize quality of life.

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