Retrospective Analysis of Pediatric Optic Pathway Gliomas: Impact of NF1 Status on Visual and Structural Outcomes.

Abstract

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. Retrospective clinical cohort study. 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%). 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. Best-corrected visual acuity (LogMAR) and retinal nerve fiber layer (RNFL) thickness. 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. 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.