Lee Leonard, Leung Jovi, Chan Brendan Min-Wei, Byrnes Joshua, Kim Hansoo
Centre for Applied Health Economics, School of Medicine and Dentistry, Griffith University, 1 Parklands Drive, Southport, QLD, 4215, Australia.
Faculty of Medicine and Health, University of Sydney, Sydney, Australia.
Orphanet J Rare Dis. 2025 Jun 13;20(1):305. doi: 10.1186/s13023-025-03837-7.
This systematic review sought to understand the types of quality of life or clinical outcome measures currently being utilised for autoimmune encephalitis (AE) patients to determine whether the current measured outcomes accurately represented the full disease burden of AE. This included how the measures were being used (e.g., time point utilised, as an endpoint or as a categorisation method), and what the recorded measures were. The review included all studies that fulfilled the population criteria, which included probable AE. A search conducted on the 24th of June 2024 on PubMed and Embase returned 302 studies that fulfilled the inclusion criteria. Of the 302, the most commonly used measures were the Modified Rankin Scale (mRS) being utilised in 90.07% of studies, followed by Clinical Assessment Scale in Autoimmune Encephalitis (CASE) at 15.56%, Montreal Cognitive Assessment (MoCA) at 7.95%, and finally Mini Mental State Examination (MMSE) at 4.97%. The most common phenotype in the literature was NMDA AE, with 208 studies (68.87%) investigating this cohort. There was significant heterogeneity in the application of measures, such as definitional differences (e.g., a good outcome defined as an mRS of < 1 for some papers, but < 2 for others), utilising measures at differing time points within the disease course of an individual, and the inconsistent reporting of key information, such as comorbidities that may impact the measures being used. This review found the current clinical measures do not holistically encompass the disease burden of AE, . It is recommended that future studies utilise symptom onset as a standardised time metric, and either a combination of currently available measures, or the creation or validation of new methods to capture disease burden in AE patients.
The online version contains supplementary material available at 10.1186/s13023-025-03837-7.
本系统评价旨在了解目前用于自身免疫性脑炎（AE）患者的生活质量或临床结局测量类型，以确定当前测量的结局是否准确反映了AE的全部疾病负担。这包括这些测量方法的使用方式（例如，所使用的时间点、作为终点或分类方法）以及记录的测量内容。该评价纳入了所有符合人群标准的研究，其中包括可能患有AE的患者。2024年6月24日在PubMed和Embase上进行的搜索返回了302项符合纳入标准的研究。在这302项研究中，最常用的测量方法是改良Rankin量表（mRS），90.07%的研究使用该量表，其次是自身免疫性脑炎临床评估量表（CASE），使用率为15.56%，蒙特利尔认知评估量表（MoCA）为7.95%，最后是简易精神状态检查表（MMSE），使用率为4.97%。文献中最常见的表型是N-甲基-D-天冬氨酸受体脑炎（NMDA AE），有208项研究（68.87%）对该队列进行了调查。在测量方法的应用方面存在显著异质性，例如定义差异（例如，一些论文将良好结局定义为mRS<1，而另一些论文则定义为<2）、在个体疾病过程中的不同时间点使用测量方法以及关键信息（如可能影响所使用测量方法的合并症）报告不一致。本评价发现，当前的临床测量方法并未全面涵盖AE的疾病负担。建议未来的研究将症状发作作为标准化的时间指标，并使用现有测量方法的组合，或者创建或验证新的方法来评估AE患者的疾病负担。
在线版本包含可在10.1186/s13023-025-03837-7获取的补充材料。
