Silvagni Ettore, Chessa Elisabetta, Bergossi Francesca, D'Amico Maria Ester, Furini Federica, Guerrini Giulio, Cauli Alberto, Scirè Carlo Alberto, Bertsias George, Govoni Marcello, Piga Matteo, Bortoluzzi Alessandra
Department of Medical Sciences, Rheumatology Unit, University of Ferrara and Azienda Ospedaliero-Universitaria S. Anna, Cona (Ferrara), Italy.
Rheumatology Unit, University Clinic and Azienda Ospedaliero-Universitaria of Cagliari, Cagliari, Italy.
Rheumatology (Oxford). 2021 Dec 24;61(1):8-23. doi: 10.1093/rheumatology/keab324.
Although neuropsychiatric involvement in SLE (NPSLE) is one of the most complex and troubling manifestations of the disease, validated outcome instruments to be used as sensitive endpoints in controlled clinical trials are lacking. We performed a systematic literature review (SLR) to identify outcome measurement instruments and domains used to assess NPSLE.
The Preferred Reporting Items for Systematic Reviews and Meta-Analysis guidelines were used. Articles available in English (1967-2020), listed in PubMed, Embase, PsycINFO, Cochrane Library and the EULAR outcome measures library were screened. All domains and outcome measurement instruments were characterized according to the OMERACT Filter 2.1, considering core areas (manifestations/abnormalities, life impact, death/lifespan, societal/resource use) and contextual factors.
Of 3392 abstracts evaluated, 83 studies were included in the SLR (15 974 patients, females 89.9%). Eligible studies included domains and instruments pertinent to all core areas defined by the OMERACT, except for 'societal/resource use'. The most common core areas were 'manifestations/abnormalities', covering 10 domains pertinent to laboratory and instrumental markers, indexes and neuropsychiatric dimension (cognitive, neurologic and psychiatric field), and 'life impact', covering 7 domains related to physical function (from both the perspective of the patient and the physician), pain and quality of life.
Our study revealed great heterogeneity in the instruments derived from populations with NPSLE and none of these had high-quality evidence. This supports the need to develop and further validate a core domain set and outcome measurement instruments to promote clinical research in this field, enhancing comparability across studies.
尽管神经精神性狼疮（NPSLE）是该疾病最复杂且棘手的表现之一，但缺乏可在对照临床试验中用作敏感终点的有效结局评估工具。我们进行了一项系统文献综述（SLR），以确定用于评估NPSLE的结局测量工具和领域。
采用系统评价和Meta分析的首选报告项目指南。筛选了在PubMed、Embase、PsycINFO、Cochrane图书馆和欧洲抗风湿病联盟（EULAR）结局测量图书馆中列出的英文文章（1967 - 2020年）。根据OMERACT Filter 2.1对所有领域和结局测量工具进行了特征描述，考虑了核心领域（表现/异常、生活影响、死亡/寿命、社会/资源利用）和背景因素。
在评估的3392篇摘要中，83项研究纳入了SLR（15974例患者，女性占89.9%）。符合条件的研究包括与OMERACT定义的所有核心领域相关的领域和工具，但不包括“社会/资源利用”。最常见的核心领域是“表现/异常”，涵盖与实验室和仪器标记、指标以及神经精神维度（认知、神经和精神领域）相关的10个领域，以及“生活影响”，涵盖与身体功能（从患者和医生两个角度）、疼痛和生活质量相关的7个领域。
我们的研究揭示了源自NPSLE患者群体的工具存在很大异质性，且这些工具均缺乏高质量证据。这支持了开发并进一步验证核心领域集和结局测量工具的必要性，以促进该领域的临床研究，提高研究间的可比性。
