Journal
NEUROLOGY
Volume 81, Issue 21, Pages S15-S24Publisher
LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1212/01.wnl.0000435745.95155.b8
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Funding
- Children's Tumor Foundation
- Department of Defense [W81XWH-12-1-0155, W81XWH-05-1-0615, W81XWH-08-1-0051]
- Thrasher Research Fund
- Sarcoma Alliance for Research through Collaboration
- Pediatric Low Grade Astrocytoma Foundation
- Bayer
- Children's Discovery Institute
- NIH [NR009651-01, UL1RR031988/UL1TR000075, NS065547, NS072916, NCT00879034]
- Gilbert Family Neurofibromatosis Institute
- Novartis Pharmaceuticals Australia
- Children's Tumor Foundation of Australia
- NICHD [UO1HD068541-01]
- European Neurofibromatosis Association
- UCSD
- Johns Hopkins University
- Memorial Sloan-Kettering Cancer Center
- Cleveland Clinic Foundation
- University of Minnesota
- Biomarin
- MD Anderson Cancer Center
- University of Toronto
- Dana Farber Cancer Institute
- University of Chicago
- National Brain Tumor Society
- James S. McDonnell Foundation
- Department of Defense
- National Cancer Institute [CA136573, CA141549, CA160882]
- Children's Discovery Institute [MC-II-2012-212]
- National Cancer Foundation Children's Oncology Group
- University of Alabama at Birmingham Research Foundation
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Objective: The goal of the Response Evaluation in Neurofibromatosis and Schwannomatosis Visual Outcomes Committee is to define the best functional outcome measures for future neurofibromatosis type 1 (NF1)-associated optic pathway glioma (OPG) clinical trials. Methods: The committee considered the components of vision, other ophthalmologic parameters affected by OPG, potential biomarkers of visual function, and quality of life measures to arrive at consensus-based, evidence-driven recommendations for objective and measurable functional endpoints for OPG trials. Results: Visual acuity (VA) assessments using consistent quantitative testing methods are recommended as the main functional outcome measure for NF1-OPG clinical trials. Teller acuity cards are recommended for use as the primary VA endpoint, and HOTV as a secondary endpoint once subjects are old enough to complete it. The optic disc should be assessed for pallor, as this appears to be a contributory variable that may affect the interpretation of VA change over time. Given the importance of capturing patient-reported outcomes in clinical trials, evaluating visual quality of life using the Children's Visual Function Questionnaire as a secondary endpoint is also proposed. Conclusions: The use of these key functional endpoints will be essential for evaluating the efficacy of future OPG clinical trials.
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