title: Functional outcome measures for NF1-associated optic pathway glioma clinical trials.
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.
authors.affiliation: 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.
authors.forename: Michael J
authors.initials: MJ
authors.lastname: Fisher
authors.forename: Robert A
authors.initials: RA
authors.lastname: Avery
authors.forename: Jeffrey C
authors.initials: JC
authors.lastname: Allen
authors.forename: Simone L
authors.initials: SL
authors.lastname: Ardern-Holmes
authors.forename: Larissa T
authors.initials: LT
authors.lastname: Bilaniuk
authors.forename: Rosalie E
authors.initials: RE
authors.lastname: Ferner
authors.forename: David H
authors.initials: DH
authors.lastname: Gutmann
authors.forename: Robert
authors.initials: R
authors.lastname: Listernick
authors.forename: Staci
authors.initials: S
authors.lastname: Martin
authors.forename: Nicole J
authors.initials: NJ
authors.lastname: Ullrich
authors.forename: Grant T
authors.initials: GT
authors.lastname: Liu
mesh_terms: D002648:Child; D002986:Clinical Trials as Topic / Q000379:methods / Q000592:standards*; D032921:Consensus; D006801:Humans; D009456:Neurofibromatosis 1 / Q000150:complications / Q000628:therapy*; D020339:Optic Nerve Glioma / Q000150:complications / Q000209:etiology / Q000628:therapy*; D016896:Treatment Outcome; D014786:Vision Disorders / Q000175:diagnosis / Q000209:etiology / Q000628:therapy*; D014787:Vision Tests / Q000379:methods / Q000592:standards*