# Biomarkers of Vision Loss in Children with Optic Pathway Gliomas

> **NIH NIH R01** · CHILDREN'S HOSP OF PHILADELPHIA · 2021 · $640,762

## Abstract

Project Summary
 Low-grade gliomas are the most common brain tumor in children and frequently involve the optic nerve,
chiasm, and tract—so they are referred to as optic pathway gliomas (OPGs). OPGs cause vision loss, typically
between 1 and 8 years of age, resulting in lifelong disability as well as reduced academic and vocational
achievement. The long term goal of this research proposal is to improve the visual outcomes and clinical
management of children with OPGs. The primary objective of this proposal is to determine if optical coherence
tomography (OCT) measures of circumpapillary retinal nerve fiber layer (cpRNFL) and ganglion cell – inner
plexiform layer (GCIPL) thickness are accurate biomarkers for vision (visual acuity and visual field). The
primary hypothesis is that the magnitude and location of cpRNFL and GCIPL thickness will be tightly correlated
to visual function and, based on previously published data, both of these OCT measures will decline before
visual function declines, thereby identifying an optimal treatment window. The secondary hypothesis is that
children who recover vision while being treated for their OPG will demonstrate specific photophic negative
response (PhNR), visual evoked potential (VEP), diffusion tensor imaging (DTI) and volumetric MRI
characteristics as compared to those that do not recover vision from treatment. The rationale for the proposed
research is that OCT measures provide an objective, reproducible assessment of visual pathway integrity that
will ensure consistency across centers and clinical trials regardless of the child’s age and ability to cooperate
with standard vision testing. The primary and secondary hypotheses will be tested in three specific aims: 1)
Create a structure-function model of vision loss for children with OPGs using OCT; 2) Identify the optimal
treatment window for OPGs using longitudinal OCT; and 3) Develop multimodal biomarkers to predict
treatment response and elucidate the mechanism of vision loss. The first aim builds on previously published
data and will be validated in an adequately powered multicenter cohort. Aim 2, supported by preliminary data,
will develop predictive models of impending vision loss by using traditional and innovative statistical techniques
to identify the earliest and most precise point of cpRNFL/GCIPL decline preceding vision loss—ultimately
defining the optimal treatment window. Aim 3 will determine how biomarkers (PhNR, VEP, DTI and volumetric
MRI) across the entire visual pathway evolve depending on whether the child experiences visual recovery or
visual loss after undergoing treatment for their OPG. This project will have an immediate impact on the clinical
care, visual outcomes and diagnostic monitoring of children with OPGs. This project will also provide much
needed insight into the mechanism of vision loss from OPGs and highlight future opportunities for
neuroprotective and visual restoration strategies outlined by the NEI Audacious Goals Init...

## Key facts

- **NIH application ID:** 10130379
- **Project number:** 5R01EY029687-03
- **Recipient organization:** CHILDREN'S HOSP OF PHILADELPHIA
- **Principal Investigator:** Robert Andrew Avery
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $640,762
- **Award type:** 5
- **Project period:** 2019-03-01 → 2024-02-29

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10130379

## Citation

> US National Institutes of Health, RePORTER application 10130379, Biomarkers of Vision Loss in Children with Optic Pathway Gliomas (5R01EY029687-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10130379. Licensed CC0.

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