Shoop-Worrall Stephanie J W, Hyrich Kimme L, Wedderburn Lucy R, Thomson Wendy, Geifman Nophar
Centre for Health Informatics, Manchester Academic Health Sciences Centre, University of Manchester, Manchester, UK.
Centre for Epidemiology Versus Arthritis, Manchester Academic Health Sciences Centre, University of Manchester, Manchester, UK.
Lancet Rheumatol. 2020 Dec 4;3(2):e111-e121. doi: 10.1016/S2665-9913(20)30269-1. eCollection 2021 Feb.
Juvenile idiopathic arthritis (JIA) is a heterogeneous disease, the signs and symptoms of which can be summarised with use of composite disease activity measures, including the clinical Juvenile Arthritis Disease Activity Score (cJADAS). However, clusters of children and young people might experience different global patterns in their signs and symptoms of disease, which might run in parallel or diverge over time. We aimed to identify such clusters in the 3 years after a diagnosis of JIA. The identification of these clusters would allow for a greater understanding of disease progression in JIA, including how physician-reported and patient-reported outcomes relate to each other over the JIA disease course.
In this multicentre prospective longitudinal study, we included children and young people recruited before Jan 1, 2015, to the Childhood Arthritis Prospective Study (CAPS), a UK multicentre inception cohort. Participants without a cJADAS score were excluded. To assess groups of children and young people with similar disease patterns in active joint count, physician's global assessment, and patient or parental global evaluation, we used latent profile analysis at initial presentation to paediatric rheumatology and multivariate group-based trajectory models for the following 3 years. Optimal models were selected on the basis of a combination of model fit, clinical plausibility, and model parsimony.
Between Jan 1, 2001, and Dec 31, 2014, 1423 children and young people with JIA were recruited to CAPS, 239 of whom were excluded, resulting in a final study population of 1184 children and young people. We identified five clusters at baseline and six trajectory groups using longitudinal follow-up data. Disease course was not well predicted from clusters at baseline; however, in both cross-sectional and longitudinal analyses, substantial proportions of children and young people had high patient or parent global scores despite low or improving joint counts and physician global scores. Participants in these groups were older, and a higher proportion of them had enthesitis-related JIA and lower socioeconomic status, compared with those in other groups.
Almost one in four children and young people with JIA in our study reported persistent, high patient or parent global scores despite having low or improving active joint counts and physician's global scores. Distinct patient subgroups defined by disease manifestation or trajectories of progression could help to better personalise health-care services and treatment plans for individuals with JIA.
Medical Research Council, Versus Arthritis, Great Ormond Street Hospital Children's Charity, Olivia's Vision, and National Institute for Health Research.
青少年特发性关节炎（JIA）是一种异质性疾病，其体征和症状可通过综合疾病活动度测量进行总结，包括临床青少年关节炎疾病活动评分（cJADAS）。然而，儿童和青少年群体在疾病体征和症状方面可能会经历不同的整体模式，这些模式可能随时间并行或出现分歧。我们旨在确定JIA诊断后3年内的此类集群。识别这些集群将有助于更深入地了解JIA的疾病进展，包括在JIA病程中医生报告的结果和患者报告的结果之间的相互关系。
在这项多中心前瞻性纵向研究中，我们纳入了2015年1月1日前招募到儿童关节炎前瞻性研究（CAPS，一项英国多中心起始队列研究）中的儿童和青少年。没有cJADAS评分的参与者被排除。为了评估在活跃关节计数、医生整体评估以及患者或家长整体评估方面具有相似疾病模式的儿童和青少年群体，我们在初次就诊于儿科风湿病科时使用了潜在类别分析，并在接下来的3年中使用了基于多变量组的轨迹模型。基于模型拟合、临床合理性和模型简约性的综合考虑选择最佳模型。
在2001年1月1日至2014年12月3日期间，1423名患有JIA的儿童和青少年被招募到CAPS，其中239名被排除，最终研究人群为1184名儿童和青少年。我们在基线时识别出五个集群，并使用纵向随访数据确定了六个轨迹组。基线时的集群并不能很好地预测疾病进程；然而，在横断面和纵向分析中，尽管关节计数较低或有所改善且医生整体评分较低，但仍有相当比例的儿童和青少年患者或家长的整体评分较高。与其他组相比，这些组中的参与者年龄较大，且患附着点炎相关JIA的比例更高，社会经济地位更低。
在我们的研究中，近四分之一的JIA儿童和青少年报告称，尽管活跃关节计数较低或有所改善且医生整体评分较低，但患者或家长的整体评分持续较高。根据疾病表现或进展轨迹定义的不同患者亚组有助于更好地为JIA患者个性化医疗服务和治疗计划。
医学研究理事会、对抗关节炎组织、大奥蒙德街儿童医院慈善机构、奥利维亚愿景基金会和国家卫生研究院。
