Novel visual outcome measures in optic neuritis and multiple sclerosis

描述（由申请人提供）：该申请由Laura J. Balcer, m.d., m.s.c.e.指导，扩展了以患者为导向的研究项目，Laura J. Balcer是一名成熟的临床研究者，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博士的研究已经证明了通过光学相干断层扫描（OCT）对MS患者的低对比度信敏度、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）结果评估中的作用，这是MS患者急性脱髓鞘和轴突/神经元损失的模型，也是检查神经保护疗法的理想目标。研究计划将实现以下目标：

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.