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风湿性疾病观察性纵向预后研究的分析方法:一项综述及基于临床队列的青少年皮肌炎患者实例分析

Methods for analyzing observational longitudinal prognosis studies for rheumatic diseases: a review & worked example using a clinic-based cohort of juvenile dermatomyositis patients.

作者信息

Lim Lily Siok Hoon, Pullenayegum Eleanor, Moineddin Rahim, Gladman Dafna D, Silverman Earl D, Feldman Brian M

机构信息

Children's Hospital Research Institute of Manitoba, University of Manitoba, Winnipeg, Canada.

Department of Pediatrics, University of Manitoba, Winnipeg, Canada.

出版信息

Pediatr Rheumatol Online J. 2017 Mar 29;15(1):18. doi: 10.1186/s12969-017-0148-2.

DOI:10.1186/s12969-017-0148-2
PMID:28356102
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5371187/
Abstract

Most outcome studies of rheumatic diseases report outcomes ascertained on a single occasion. While single assessments are sufficient for terminal or irreversible outcomes, they may not be sufficiently informative if outcomes change or fluctuate over time. Consequently, longitudinal studies that measure non-terminal outcomes repeatedly afford a better understanding of disease evolution.Longitudinal studies require special analytic methods. Newer longitudinal analytic methods have evolved tremendously to deal with common challenges in longitudinal observational studies. In recent years, an increasing number of studies have used longitudinal design. This review aims to help readers understand and apply the findings from longitudinal studies. Using a cohort of children with juvenile dermatomyositis (JDM), we illustrate how to study evolution of disease activity in JDM using longitudinal methods.

摘要

大多数风湿性疾病的结局研究报告的是单次确定的结局。虽然单次评估对于终末或不可逆结局就足够了,但如果结局随时间变化或波动,它们可能提供的信息就不够充分。因此,反复测量非终末结局的纵向研究能更好地了解疾病演变。纵向研究需要特殊的分析方法。更新的纵向分析方法已经有了巨大发展,以应对纵向观察性研究中的常见挑战。近年来,越来越多的研究采用了纵向设计。本综述旨在帮助读者理解和应用纵向研究的结果。我们以一组青少年皮肌炎(JDM)患儿为例,说明如何使用纵向方法研究JDM疾病活动的演变。

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