Michael J Fisher1, 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. 1. From the Division of Oncology (M.J.F.), Neuroradiology Section, Department of Radiology (L.T.B.), and Neuro-Ophthalmology Service (G.T.L.), The Children's Hospital of Philadelphia; Department of Pediatrics (M.J.F.) and Departments of Neurology and Ophthalmology (G.T.L.), The Perelman School of Medicine at the University of Pennsylvania, Philadelphia; Departments of Neurology, Ophthalmology, and Pediatrics (R.A.A.), Gilbert Family Neurofibromatosis Institute, Children's National Medical Center, Washington, DC; Departments of Pediatrics and Neurology (J.C.A.), NYU Cancer Institute, NYU Langone Medical Center, New York, NY; Children's Hospital at Westmead Clinical School (S.L.A.-H.), The University of Sydney, Australia; Department of Neurology (S.L.A.-H.), The Children's Hospital at Westmead, Sydney, Australia; University of Pennsylvania School of Medicine (L.T.B.), Philadelphia; Department of Neurology (R.E.F.), Guy's and St. Thomas' NHS Foundation Trust and Institute of Psychiatry, King's College London; Department of Neurology (D.H.G.), Washington University School of Medicine, St. Louis, MO; Department of Pediatrics (R.L.), Feinberg School of Medicine, Northwestern University; Ann & Robert H. Lurie Children's Hospital of Chicago (R.L.); Pediatric Oncology Branch (S.M.), National Cancer Institute, National Institutes of Health, Bethesda, MD; and Department of Neurology (N.J.U.), Boston Children's Hospital, Harvard Medical School, Boston, MA.
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.
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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