Leveraging Visual Outcome Measures to Advance Therapy Development in Neuroimmunologic Disorders.
Leveraging Visual Outcome Measures to Advance Therapy Development in Neuroimmunologic Disorders.
复制标题
DOI：
10.1212/nxi.0000000000001126
复制
发表时间：
2022-03
期刊：
影响因子：
--
通讯作者：
IMSVISUAL
中科院分区：
文献类型：
--
作者：
Graves JS;Oertel FC;Van der Walt A;Collorone S;Sotirchos ES;Pihl-Jensen G;Albrecht P;Yeh EA;Saidha S;Frederiksen J;Newsome SD;Paul F;IMSVISUAL
视觉系统在评估神经轴突损伤方面提供了无与伦比的精确度。随着大多数多发性硬化症(MS)患者经历传入和传出视觉功能障碍，捕捉这些缺陷的结果测量提供了对神经轴突损伤的洞察，即使在那些残疾程度最低的患者中也是如此。视觉测量是临床试验的理想选择，通常价格低廉，易于获得，且可重复性好。量化视力、视野、视觉生活质量和电生理参数可以评估功能，而光学相干断层扫描(OCT)可以可靠地测量前部传入视觉通路的结构完整性。眼球运动生物识别技术不断进步，对注视、平稳追踪和眼球运动异常的离散测量已准备好纳入未来的MS进展试验。视觉结果可以跟踪神经轴突损伤，并有助于将MS与诸如神经脊髓炎视神经谱系障碍(NMOSD)或髓鞘少突胶质细胞糖蛋白抗体相关疾病(MOGAD)等疾病区分开来。OCT还为病理生理学提供了独特的见解，包括在NMOSD中鉴定中心凹凹陷，可能是由于Müler细胞的损伤，Müler细胞携带大量的水通道蛋白-4通道。对于一些研究设计，成本效益比更昂贵的MRI结果更有利于视觉结果。随着治疗学的下一个前沿集中在髓鞘再生和神经保护上，视觉结果可能会成为焦点。国际MS视觉系统联盟(IMSVISUAL)的这篇综述概述了将视觉结果常规纳入MS和NMOSD试验所需的质量标准、信息学和框架，这是一个由合作、坚定的视觉科学家组成的国际社区。
The visual system offers unparalleled precision in the assessment of neuroaxonal damage. With the majority of patients with multiple sclerosis (MS) experiencing afferent and efferent visual dysfunction, outcome measures capturing these deficits provide insight into neuroaxonal injury, even in those with minimal disability. Ideal for use in clinical trials, visual measures are generally inexpensive, accessible, and reproducible. Quantification of visual acuity, visual fields, visual quality of life, and electrophysiologic parameters allows assessment of function, whereas optical coherence tomography (OCT) provides reliable measures of the structural integrity of the anterior afferent visual pathway. The technology of oculomotor biometrics continues to advance, and discrete measures of fixation, smooth pursuit, and saccadic eye movement abnormalities are ready for inclusion in future trials of MS progression. Visual outcomes allow tracking of neuroaxonal injury and aid in distinguishing MS from diseases such as neuromyelitis optica spectrum disorder (NMOSD) or myelin oligodendrocyte glycoprotein antibody–associated diseases (MOGAD). OCT has also provided unique insights into pathophysiology, including the identification of foveal pitting in NMOSD, possibly from damage to Müller cells, which carry an abundance of aquaporin-4 channels. For some study designs, the cost-benefit ratio favors visual outcomes over more expensive MRI outcomes. With the next frontier of therapeutics focused on remyelination and neuroprotection, visual outcomes are likely to take center stage. As an international community of collaborative, committed, vision scientists, this review by the International MS Visual System Consortium (IMSVISUAL) outlines the quality standards, informatics, and framework needed to routinely incorporate vision outcomes into MS and NMOSD trials.