Cross-Sectional Profiling of Aqueous Humor VEGF-A Reflects the Tumor and Ocular Microenvironment in Retinoblastoma.
4 affiliations
- 1The Vision Center, Children's Hospital Los Angeles, Los Angeles, California, United States.
- 2USC Roski Eye Institute, University of Southern California Keck School of Medicine, Los Angeles, California, United States.
- 3Division of Hematology Oncology, Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, California, United States.
- 4University of Southern California Keck School of Medicine, Los Angeles, California, United States.
Abstract
Purpose
Vascular endothelial growth factor A (VEGF-A) is a key mediator of tumor angiogenesis. This cross-sectional study quantified aqueous humor (AH) VEGF-A in retinoblastoma (RB) eyes and retrospectively evaluated its association with clinical features.Methods
This study analyzed 60 AH samples: 54 from 49 RB eyes and 6 from age-matched controls with congenital cataract. From pretreatment RB eyes, six samples were collected prior to globe-salvaging treatment and five immediately after primary enucleation (PE). From post-treatment eyes, 12 samples were collected at secondary enucleation (SE), 12 during active treatment (TX), and 19 at 6 months after treatment (END). VEGF-A was measured using the Olink Proximity Extension Assay and reported as absolute concentrations (pg/mL).Results
VEGF-A concentrations (median and interquartile range [IQR]) were 259 (95% confidence interval [CI] = 140-423, IQR = 115-568) in the RB cohort, compared with 149 (95% CI = 39.8-318, IQR = 61.3-208) in cataract controls. Post-treatment levels were significantly lower than pretreatment levels (median = 182, 95% CI = 121-310, IQR = 110-423 vs. median = 1380, 95% CI = 278-18,100, IQR = 467-12,400, P < 0.001). Among treated eyes, SE samples had higher VEGF-A than END samples (median = 506, 95% CI = 120-1010, IQR = 207-926 vs. median = 129, 95% CI = 79.5-212, IQR = 79.5-212, P = 0.007).Conclusions
AH liquid biopsy was a high-yield source of VEGF-A in RB. VEGF-A concentrations were highest during primary enucleation and normalized after treatment completion. These findings support that aqueous VEGF-A reflects the intraocular microenvironment, warranting further validation to improve our understanding of its role in disease progression in RB.
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BibTeX
@article{lin2026crosssectional,
title = {{Cross-Sectional Profiling of Aqueous Humor VEGF-A Reflects the Tumor and Ocular Microenvironment in Retinoblastoma.}},
author = {Michelle Lin and Chen-Ching Peng and Mark Reid and Elaine Huang and Nerea Goni and Kaitlyn Phung and Yilin Chen and Drishti Pandya and Brianne Brown and Bibiana J Reiser and Rachana Shah and Jesse L Berry and Liya Xu},
journal = {Investigative ophthalmology \& visual science},
year = {2026},
month = oct,
doi = {10.1167/iovs.67.12.23},
url = {https://europepmc.org/article/MED/42872949},
note = {CC BY},
}
RIS
TY - JOUR
TI - Cross-Sectional Profiling of Aqueous Humor VEGF-A Reflects the Tumor and Ocular Microenvironment in Retinoblastoma.
AU - Michelle Lin
AU - Chen-Ching Peng
AU - Mark Reid
AU - Elaine Huang
AU - Nerea Goni
AU - Kaitlyn Phung
AU - Yilin Chen
AU - Drishti Pandya
AU - Brianne Brown
AU - Bibiana J Reiser
AU - Rachana Shah
AU - Jesse L Berry
AU - Liya Xu
PY - 2026
DA - 2026/10/01
JO - Investigative ophthalmology & visual science
DO - 10.1167/iovs.67.12.23
UR - https://europepmc.org/article/MED/42872949
AB - Purpose Vascular endothelial growth factor A (VEGF-A) is a key mediator of tumor angiogenesis. This cross-sectional study quantified aqueous humor (AH) VEGF-A in retinoblastoma (RB) eyes and retrospectively evaluated its association with clinical features.Methods This study analyzed 60 AH samples: 54 from 49 RB eyes and 6 from age-matched controls with congenital cataract. From pretreatment RB eyes, six samples were collected prior to globe-salvaging treatment and five immediately after primary enucleation (PE). From post-treatment eyes, 12 samples were collected at secondary enucleation (SE), 12 during active treatment (TX), and 19 at 6 months after treatment (END). VEGF-A was measured using the Olink Proximity Extension Assay and reported as absolute concentrations (pg/mL).Results VEGF-A concentrations (median and interquartile range [IQR]) were 259 (95% confidence interval [CI] = 140-423, IQR = 115-568) in the RB cohort, compared with 149 (95% CI = 39.8-318, IQR = 61.3-208) in cataract controls. Post-treatment levels were significantly lower than pretreatment levels (median = 182, 95% CI = 121-310, IQR = 110-423 vs. median = 1380, 95% CI = 278-18,100, IQR = 467-12,400, P < 0.001). Among treated eyes, SE samples had higher VEGF-A than END samples (median = 506, 95% CI = 120-1010, IQR = 207-926 vs. median = 129, 95% CI = 79.5-212, IQR = 79.5-212, P = 0.007).Conclusions AH liquid biopsy was a high-yield source of VEGF-A in RB. VEGF-A concentrations were highest during primary enucleation and normalized after treatment completion. These findings support that aqueous VEGF-A reflects the intraocular microenvironment, warranting further validation to improve our understanding of its role in disease progression in RB.
SN - 0146-0404
SN - 1552-5783
ER -