Functional outcome measures for NF1-associated optic pathway glioma clinical trials

Fisher, M. J.; Avery, R. A.; Allen, J. C.; Ardern-Holmes, S. L.; Bilaniuk, L. T.; Ferner, R. E.; Gutmann, D. H.; Listernick, R.; Martin, S.; Ullrich, N. J.; Liu, G. T.

Neurology. 2013 Nov 28; 81(21 Suppl 1):S15-24


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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