Novel visual outcome measures in optic neuritis and multiple sclerosis

视神经炎和多发性硬化症的新视觉结果测量

• 批准号: 7609034
• 负责人: LAURA BALCER
• 金额: $ 15.83万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-04-01 至 2012-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7609034
• 关键词: Acute; Address; Adult; Biological Markers; Biological Models; Blindness; Clinical Investigator; Clinical Trials; Contrast Sensitivity; Data Analyses; Data Set; Demyelinations; Development; Doctor of Medicine; Educational Status; Eye; Functional disorder; Future; Hand; Head; Image; Investigation; Letters; Longitudinal Studies; Manuscripts; Measures; Mentors; Methods; Modeling; Multiple Sclerosis; Natural History; Neurology; Neuroprotective Agents; Optic Neuritis; Optical Coherence Tomography; Outcome; Outcome Assessment; Outcome Measure; Patient Self-Report; Patients; Performance; Preparation; Public Health; Questionnaires; Recording of previous events; Relapse; Research; Research Design; Role; Scientist; Series; Testing; Therapeutic; Therapeutic Intervention; Thick; Vision; Vision research; Visual; Work; axonal degeneration; cohort; design; flexibility; health related quality of life; improved; macula; middle age; natalizumab; neuron loss; next generation; novel; patient oriented research; patient population; programs; retinal nerve fiber layer; treatment effect; treatment strategy; treatment trial; vision development

DESCRIPTION (provided by applicant): This application proposes expanded programs of patient-oriented research and mentoring by Laura J. Balcer, M.D., M.S.C.E., an established clinical investigator whose work has focused on the development of visual outcomes for clinical trials in multiple sclerosis (MS). Dr. Balcer's research has demonstrated validity for low-contrast letter acuity and for retinal nerve fiber layer (RNFL) thickness and macular volume by optical coherence tomography (OCT) in MS, but has not established roles for these parameters in outcomes assessment for acute optic neuritis (ON), a model for acute demyelization and axonal/neuronal loss in MS and an ideal target for examining neuroprotective therapies. The research plan will accomplish the following aims: Specific Aim 1: Refine and validate low-contrast letter acuity and OCT as new visual outcomes for clinical trials in MS, and in acute ON as a model system for future testing of neuroprotective agents. Specific Aim 2: Determine how new visual outcomes, low-contrast letter acuity and OCT, reflect vision-specific and overall health-related quality of life in longitudinal studies of MS and ON, and examine the validity and usefulness of a new 10-ltem Neuro-Ophthalmic Supplement to the 25-ltem NEI Visual Functioning Questionnaire (NEI-VFQ-25) in these patient populations. These investigations will further define potential roles for new measures of low-contrast letter acuity and OCT in MS clinical trials, and will apply paradigms from MS vision research to the study of acute ON as a more specific model system for assessment of natural history and neuroprotective treatment trial outcomes. Participation by mentees in refinement of study design, performance of hands-on testing, data analysis, and manuscript preparation will be a central focus of the candidate's mentoring plan. These proposed new projects have been designed and selected to facilitate trainee involvement, and are sufficiently flexible and varied in scope to incorporate mentees in Neurology at all levels of training. Public Health Statement: MS and optic neuritis are leading causes of visual loss among young and middle-aged adults in the U.S. This work will provide support for additional research and for mentoring of beginning clinician scientists in the development of improved testing methods for vision in these conditions.

描述（由申请人提供）：本申请提出了以患者为中心的研究扩展计划，并由医学博士、理学硕士 Laura J. Balcer 进行指导，她是一位知名的临床研究人员，其工作重点是开发多发性硬化症 (MS) 临床试验的视觉结果。 Balcer 博士的研究通过光学相干断层扫描 (OCT) 证明了多发性硬化症中低对比度字母敏锐度、视网膜神经纤维层 (RNFL) 厚度和黄斑体积的有效性，但尚未确定这些参数在急性视神经炎 (ON) 结局评估中的作用，急性视神经炎 (ON) 是多发性硬化症急性脱髓鞘和轴突/神经元损失的模型，也是检查神经保护作用的理想目标 疗法。该研究计划将实现以下目标： 具体目标 1：细化和验证低对比度字母敏锐度和 OCT 作为新的视觉结果 MS 和急性 ON 的临床试验作为未来神经保护剂测试的模型系统。 具体目标 2：确定新的视觉结果、低对比度字母敏锐度和 OCT 如何反映 在 MS 和 ON 的纵向研究中评估视觉特异性和整体健康相关的生活质量，并检查新的 10 项神经眼科补充对 25 项 NEI 视觉功能问卷 (NEI-VFQ-25) 在这些患者群体中的有效性和实用性。 这些研究将进一步确定低对比度字母敏锐度和 OCT 新测量方法在多发性硬化症临床试验中的潜在作用，并将把多发性硬化症视力研究的范式应用到急性视神经炎的研究中，作为评估自然史和神经保护治疗试验结果的更具体的模型系统。 受指导者参与研究设计的完善、实践测试的表现、数据分析和手稿准备将是候选人指导计划的核心重点。这些拟议的新项目的设计和选择是为了促进学员参与，并且具有足够的灵活性和多样化的范围，可以将学员纳入神经病学各个级别的培训中。 公共卫生声明：多发性硬化症和视神经炎是美国年轻人和中年人视力丧失的主要原因。这项工作将为更多研究提供支持，并指导初级临床科学家开发改进的视力测试方法。