对Sjögren疾病随机试验中临床试验设计和结果测量的系统回顾。

IF 3.6 2区 医学 Q2 RHEUMATOLOGY
Maxime Beydon, Yann Nguyen, Rachael Gordon, Nathan Foulquier, Coralie Bouillot, Katherine M Hammitt, Simon J Bowman, Xavier Mariette, Divi Cornec, Sara S McCoy, Raphaèle Seror
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引用次数: 0

摘要

目的:系统回顾目前所有用于Sjögren's disease (SjD)临床试验的SjD相关仪器。方法:系统检索Medline (Pubmed)和EMBASE,检索时间为2002年至2023年3月的所有随机对照试验(rct),采用人工方法和人工智能软件(BIBOT)。我们提取了所有用作主要或次要结局的工具,并评估研究是否达到或没有改善结局。我们还根据最近初步确定的结果域对这些工具进行了分类。结果:在5420篇文献中,纳入了60项rct,这些rct要么关注整体疾病表现(53%),要么关注单个器官/症状(例如:干眼(17%)、口干(15%)、疲劳(12%)或肺功能(3%)。主要结局包括口腔或眼干、患者报告结局(PRO)、全身活动和其他结局(7%)。常用仪器有esdai、ESSPRI、Schirmer试验、无刺激唾液流量、IgG水平。11项研究将ESSDAI作为主要结局,45%的研究达到显著性,而16项将ESSDAI作为次要结局的研究均未达到显著性。在34项研究中,PROs是主要结局。腺体功能测量结果各不相同,未受刺激的唾液流量是最常见的测量结果。生活影响更频繁地被评估为次要结果。只有两项研究关注生物活性。结论:我们的综述强调了SjD在设计和结果上的异质性。近年来,PROs和综合结果的使用有所增加,突出了从客观干燥测量到更全面的以患者为中心的结果的转变。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
A Systematic Review of Clinical Trial Designs and Outcome Measures in Sjögren Disease Randomized Controlled Trials.

Objective: To systematically review all existing Sjögren disease (SjD)-related instruments reported in clinical trials for SjD.

Methods: We systematically searched Medline (PubMed) and EMBASE between January 2002 and March 2023 to identify all randomized controlled trials (RCTs) using both a manual approach and artificial intelligence software (Bibliography BOT). We extracted all the instruments used as primary or secondary outcomes and assessed whether the study succeeded in improving the outcome. We also classified the instruments according to the recently defined preliminary outcome domains.

Results: Among 5420 references, 60 RCTs were included, focusing either on overall disease manifestations (53%) or on a single organ/symptom (eg, dry eyes [17%], xerostomia [15%], fatigue [12%], or pulmonary function [3%]). Primary outcomes included measures of oral or ocular dryness, patient-reported outcomes (PROs), systemic activity, and other outcomes. Common instruments used were European Alliance of Associations for Rheumatology (EULAR) Sjögren Syndrome Disease Activity Index (ESSDAI), EULAR Sjögren Syndrome Patient-Reported Index, Schirmer-I test for unstimulated salivary flow, and IgG levels. ESSDAI was a primary outcome in 11 studies, with 45% of studies reaching significance, whereas none of the 16 studies with ESSDAI as a secondary outcome reached significance. PROs were the primary outcome in 34 studies. Glandular function measurements varied, with unstimulated salivary flow as the most commonly measured outcome. Life impact was assessed more frequently as a secondary outcome. Only 2 studies focused on biological activity.

Conclusion: Our review highlighted the heterogeneity of SjD RCTs in both the study designs and outcomes. The use of PROs and composite outcomes has increased in recent years, highlighting a shift from objective dryness measures to more holistic patient-centered outcomes.

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来源期刊
Journal of Rheumatology
Journal of Rheumatology 医学-风湿病学
CiteScore
6.50
自引率
5.10%
发文量
285
审稿时长
1 months
期刊介绍: The Journal of Rheumatology is a monthly international serial edited by Earl D. Silverman. The Journal features research articles on clinical subjects from scientists working in rheumatology and related fields, as well as proceedings of meetings as supplements to regular issues. Highlights of our 41 years serving Rheumatology include: groundbreaking and provocative editorials such as "Inverting the Pyramid," renowned Pediatric Rheumatology, proceedings of OMERACT and the Canadian Rheumatology Association, Cochrane Musculoskeletal Reviews, and supplements on emerging therapies.
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