The CLN3 Disease Staging System: A new tool for clinical research in Batten disease
The CLN3 Disease Staging System: A new tool for clinical research in Batten disease
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DOI：
10.1212/wnl.0000000000009454
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发表时间：
2020-06-09
期刊：
影响因子：
9.9
通讯作者：
Mink, Jonathan W.
中科院分区：
文献类型：
--
作者：
Masten, Margaux C.;Williams, Justin D.;Mink, Jonathan W.
目的开发一种疾病特异性分期系统CLN3疾病和检验的假设，CLN3疾病的显着和离散的临床特征，可用于定义疾病阶段的分析数据从一个18年之久的自然history study.MethodsA提出的分期系统，CLN3分期系统（CLN3SS），是基于显着和临床意义的终点。确定了分期与年龄、分期与统一Batten疾病评定量表（UBDRS）身体严重程度评分、分期与UBDRS能力损害子量表评分之间的关系。我们用t检验，以确定是否阶段是显着不同的年龄和scores.ResultsData的基础上，从322评价108个人进行了分析。根据年龄和严重程度评分，各阶段之间存在显著差异。对于个人的纵向数据，没有个人恢复到一个不太严重的阶段随着时间的推移.ConclusionThe CLN3SS是一种疾病特异性分期系统，可用于根据年龄和疾病的严重程度将个人分为特定的阶层。CLN3SS在队列分层的临床试验中具有潜在应用。
ObjectiveTo develop a disease-specific staging system for CLN3 disease and to test the hypothesis that salient and discrete clinical features of CLN3 disease may be used to define disease stages by analyzed data from an 18-year-long natural history study.MethodsA proposed staging system, the CLN3 Staging System (CLN3SS), was based on salient and clinically meaningful endpoints. The relationships between stage and age, stage and Unified Batten Disease Rating Scale (UBDRS) physical severity score, and stage and UBDRS capability impairment subscale scores were determined. We used t tests to determine whether the stages were significantly different from each other on the basis of age and scores.ResultsData were analyzed from 322 evaluations in 108 individuals. There were significant differences among the stages based on age and severity scores. For individuals with longitudinal data, no individual reverted to a less severe stage over time.ConclusionThe CLN3SS is a disease-specific staging system that can be used to classify individuals into specific strata based on age and disease severity. The CLN3SS has potential applications in clinical trials for cohort stratification.