用于 NF1 相关视神经胶质瘤临床试验的功能结局测量。
Functional outcome measures for NF1-associated optic pathway glioma clinical trials.
机构信息
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.
出版信息
Neurology. 2013 Nov 19;81(21 Suppl 1):S15-24. doi: 10.1212/01.wnl.0000435745.95155.b8.
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.
目的
神经纤维瘤病和雪旺氏细胞瘤视觉结局委员会的目标是确定用于未来神经纤维瘤病 1 型（NF1）相关视神经胶质瘤（OPG）临床试验的最佳功能结果测量指标。
方法
委员会考虑了视力的组成部分、OPG 影响的其他眼科参数、视觉功能的潜在生物标志物以及生活质量测量指标，以达成基于共识和证据的建议，为 OPG 试验确定客观和可衡量的功能终点。
结果
建议使用一致的定量测试方法评估视力（VA），作为 NF1-OPG 临床试验的主要功能结果测量指标。推荐使用 Teller 视力卡作为主要的 VA 终点，一旦受试者足够大能够完成，HOTV 则作为次要终点。应评估视盘苍白，因为这似乎是一个可能影响 VA 随时间变化的解释的贡献变量。鉴于在临床试验中捕获患者报告结果的重要性，还建议使用儿童视觉功能问卷评估视觉生活质量作为次要终点。
结论
这些关键功能终点的使用对于评估未来 OPG 临床试验的疗效至关重要。
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