Functional outcome measures for NF1-associated optic pathway glioma clinical trials
Michael J Fisher, Robert A Avery, Jeffrey C Allen, Simone L Ardern-Holmes, Larissa T Bilaniuk, Rosalie E Ferner, David H Gutmann, Robert Listernick, Staci Martin, Nicole J Ullrich, Grant T Liu, REiNS International Collaboration, Michael J Fisher, Robert A Avery, Jeffrey C Allen, Simone L Ardern-Holmes, Larissa T Bilaniuk, Rosalie E Ferner, David H Gutmann, Robert Listernick, Staci Martin, Nicole J Ullrich, Grant T Liu, REiNS International Collaboration
Abstract
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.
Trial registration: ClinicalTrials.gov NCT00879034.
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Source: PubMed