Retrospective Analysis of Pediatric Optic Pathway Gliomas: Impact of NF1 Status on Visual and Structural Outcomes.
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PURPOSE: To evaluate visual outcomes, optical coherence tomography (OCT) parameters, and treatment modalities in pediatric patients with optic pathway gliomas (OPGs) in Lebanon, comparing those with neurofibromatosis type 1 (NF1) to non-NF1 patients. DESIGN: Retrospective clinical cohort study. PARTICIPANTS: Seventy-two pediatric patients (144 eyes) diagnosed with OPGs at the American University of Beirut Medical Center between January 2000 and August 2023. The cohort included 40 NF1 eyes (27.8%) and 104 non-NF1 eyes (72.2%). METHODS: Data collected included demographics, clinical presentation, visual acuity (VA), OCT measurements, NF1 status, and detailed treatment history. Longitudinal changes in structural and functional outcomes were assessed using paired t-tests. MAIN OUTCOME MEASURES: Best-corrected visual acuity (LogMAR) and retinal nerve fiber layer (RNFL) thickness. RESULTS: Treatment was significantly more common in non-NF1 patients, including surgery (69.2% vs 5%), chemotherapy (67.3% vs 25%), and radiation (30.8% vs 5%) (all P < .001). Overall VA improved, with non-NF1 eyes showing significant improvement (LogMAR 0.28 to 0.21; P = .0077), while NF1 eyes showed a nonsignificant trend (LogMAR 0.27 to 0.18; P = .0768). RNFL thickness declined in both groups but more prominently in non-NF1 eyes (99.9 to 75.5 μm; P = .0019) compared with NF1 eyes (73.0 to 70.4 μm; P = .0202). Among non-NF1 patients, chemotherapy was associated with worse final VA (LogMAR 0.35 vs 0.06; P = .007) and more pronounced RNFL thinning. Limitations include retrospective design and incomplete baseline testing for some patients. CONCLUSIONS: NF1-associated OPGs exhibited a more indolent disease course, lower treatment rates, and preserved vision despite RNFL thinning. Non-NF1 patients underwent more aggressive treatment and demonstrated greater visual improvement, although chemotherapy was linked to poorer final outcomes. OCT serves as a valuable tool for disease monitoring, particularly in NF1 patients.