ID #349 Beyond tumor control: late effects in pediatric optic pathway glioma in an Low-middle income setting
Maya Prasad, Mili Aggarwal, Vikas Patil, Venkata Ramamohan Gollamudi, Badira Parambil, Ayushi Jain, Aekta Shah, Arpita Sahu, Kajari Bhattacharya, Amit Choudhuri, Vasundhara Patil, Amrita Guha, Vikas Singh, Prakash Shetty, Abhishek Chatterji, Archya Dasgupta, Aliasgar Moiyadi, Sridhar Epari, Tejpal Gupta, Girish ChinnaswamyAbstract
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.