Development and Validation of a Juvenile Spondyloarthritis Disease Flare Measure: Ascertaining Flare in Patients With Inactive Disease.

Development and Validation of a Juvenile Spondyloarthritis Disease Flare Measure: Ascertaining Flare in Patients With Inactive Disease.
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DOI:
10.1002/acr.24763
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发表时间:
2023-03
影响因子:
4.7
通讯作者:
Lovell D
Lovell D
中科院分区:
医学2区
文献类型:
--
作者:
Weiss PF;Brandon TG;Ryan ME;Treemarcki EB;Armendariz S;Wright TB;Godiwala C;Stoll ML;Xiao R;Lovell D

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我们的目标是开发和验证幼年脊柱性关节炎的复合疾病症状定义,该定义将接近于在治疗降级后重新启动/不重新启动系统治疗的临床决定。对接受系统治疗、生物或传统抗风湿药物(bDMARDS;cDMARDS)降级治疗的脊柱炎儿童进行回顾性图表回顾,以开发和验证闪光结果。开发(1个中心)和验证(4个中心)的独立队列收集自大型三级医疗保健系统。使用灵敏度、特异度、阳性(PPV)和阴性预测值(NPV)以及受试者工作特征(AUROC)曲线下面积来评估核心测量阈值和候选疾病病情转归,以医生对“活动性疾病”的评估加上重新开始系统治疗的标准剂量作为参考标准。在候选定义中,以下五个核心指标中有临床意义的恶化在≥3中表现最好:护理者/患者对幸福感的评估,医生对疾病活动的评估,护理者/患者对疼痛的评估,身体功能和活跃的关节计数。AUROC为0.91,PPV为87.5%,NPV为98.1%,敏感性为82.4%,特异性为98.7%。克朗巴赫的α为0.81,表明内部一致性,因素分析表明结果被测为一个结构。根据21名接受调查的儿科风湿病医生的说法,“JSpAflare”具有表面有效性。JSpAflare在验证队列中的AUROC为0.85,PPV为92.3%,NPV为96.8%。初步支持JSpAflare作为一种工具在青少年脊柱炎患者降级治疗中识别疾病闪光的有效性,并有可能在临床实践、观察性研究和治疗试验中应用。
Our objective was to develop and validate a composite disease flare definition for juvenile spondyloarthritis that would closely approximate the clinical decision made to reinitiate/not reinitiate systemic therapy after therapy de-escalation. Retrospective chart reviews of children with spondyloarthritis who underwent systemic therapy de-escalation of biologic or conventional disease-modifying antirheumatic drugs (bDMARDs; cDMARDs) were used to develop and validate the flare outcome. Independent cohorts for development (1 center) and validation (4 centers) were collected from large tertiary healthcare systems. Core measure thresholds and candidate disease flare outcomes were assessed using sensitivity, specificity, positive (PPV) and negative predictive values (NPV), and area under the receiver operating characteristic (AUROC) curve with physician assessment of “active disease” plus re-initiation of standard dose of systemic therapy as the reference standard. Of the candidate definitions, clinically meaningful worsening in ≥3 of the following five core measures performed best: caregiver/patient assessment of well-being, physician assessment of disease activity, caregiver/patient assessment of pain, physical function, and active joint count. AUROC was 0.91, PPV 87.5%, NPV 98.1%, sensitivity 82.4%, and specificity 98.7%. Cronbach’s α was 0.81, signifying internal consistency and factor analysis demonstrated the outcome measured one construct. “JSpAflare” had face validity according to 21 surveyed pediatric rheumatologists. JSpAflare had AUROC 0.85, PPV 92.3%, and NPV 96.8% in the validation cohort. There is initial support for the validity of JSpAflare as a tool to identify disease flare in juvenile spondyloarthritis patients de-escalating therapy and is potentially applicable in clinical practice, observational studies, and therapeutic trials.
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