中国专家共识:遗传性视网膜营养不良临床试验中多亮度移动性测试的规范化应用  

Chinese expert consensus on the standardized application of multi-luminance mobility test in clinical trials for inherited retinal dystrophy

作　　者：中华医学会眼科学分会眼底病学组 中国医师协会眼科医师分会眼底病学组 孙晓东 许迅[2] Fundus Disease Group of Ophthalmology Society of Chinese Medical Association;Fundus Disease Committee of Ophthalmologist Branch of Chinese Medical Doctor Association

机构地区：[1]不详 [2]国家眼部疾病临床医学研究中心上海交通大学医学院附属第一人民医院眼科,上海200080

出　　处：《中华眼底病杂志》2025年第3期169-177,共9页Chinese Journal of Ocular Fundus Diseases

摘　　要：近年来,随着对遗传性视网膜营养不良(IRD)等眼罕见病研究的深入,尤其是细胞和基因治疗技术的重大突破,为这些疾病的治疗带来了新的希望。为了有效评估IRD患者的疾病进展、创新疗法的安全性和疗效,建立标准化的视觉功能评价终点和检测方法变得至关重要。传统的最佳矫正视力(BCVA)是公认的视功能主要评价指标之一。然而,对于以视杆细胞严重损伤为主的IRD患者,这类患者多为严重低视力甚至法定盲者,BCVA这一传统指标的应用受到了挑战。为此,多亮度移动性测试(MLMT)作为一种功能性视觉评估工具应运而生。MLMT通过在不同光照条件下评估受试者导航障碍路线的能力,设立分级评价标准,客观量化光照条件对患者移动和定位能力的影响,为IRD临床试验提供了新颖且量化的视功能评估手段。尽管MLMT展示了其独特价值,但国内目前尚缺乏关于该测试应用的统一化和规范化建议,这在一定程度上限制了MLMT在实际临床研究中的广泛应用。鉴于此,中华医学会眼科学分会眼底病学组联合中国医师协会眼科医师分会眼底病学组,邀请遗传性视网膜疾病领域的临床专家、统计学专家及光学专家共同参与,调研当前MLMT的应用现状和技术特点,并制定针对IRD基因治疗药物临床试验中MLMT应用的共识性建议。本共识旨在提供一套适用于中国国情的MLMT操作规范,指导眼科及相关学科的临床实践与研究,推动我国IRD临床试验的标准化进程,从而更好地服务于IRD患者群体,并促进相关领域的发展。With the advancement of research on rare ocular diseases such as inherited retinal dystrophy(IRD)has advanced in recent years,especially breakthroughs in therapeutic approaches represented by cell and gene therapy,potential intervention strategies have emerged for these conditions.Establishing standardized endpoints and evaluation methods for visual function in patients with IRD has become crucial for assessing disease progression,safety,and therapeutic efficacy of innovative treatments.Best corrected visual acuity(BCVA)is widely recognized as one of the primary endpoints for assessing visual function.However,for IRD patients with severe rod photoreceptor dysfunction,who often present with profound low vision or even legal blindness,the applicability of BCVA as a traditional visual function indicator is limited.The multi-luminance mobility test(MLMT)has emerged as a functional visual assessment tool that evaluates the ability to navigate obstacle courses under varying illumination levels.By establishing graded evaluation standards,MLMT objectively quantifies the impact of lighting conditions on patients'nobility and spatial orientation,providing a novel quantitative tool for assessing visual function in IRD clinical trials.Currently,there is a lack of unified and standardized guidelines for the use of MLMT in China,posing challenges to its implementation in practical clinical research.To address this,Ocular Fundus Disease Society of Chinese Medical Association and Chinese Medical Doctor Association convened a multidisciplinary team comprising clinical experts in genetic retinal diseases,statisticians,and optical specialists to investigate the current applications and technical characteristics of MLMT,ultimately formulating consensus recommendations for its use as a clinical trial endpoint for IRD gene therapies.This consensus aims to provide a set of MLMT operating norms applicable to China's national conditions,guide clinical practice and research in ophthalmology and related disciplines,and promote the standard

关 键 词：遗传性视网膜营养不良 多亮度移动性测试 评价指标 临床研究 专家共识 

分 类 号：R73[医药卫生—肿瘤]