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荷兰青少年特发性关节炎患者的药物治疗模式:真实世界数据分析。

Pharmacological treatment patterns in patients with juvenile idiopathic arthritis in the Netherlands: a real-world data analysis.

机构信息

Department of Health Technology and Services Research, Faculty of Behavioural, Management and Social Sciences, Technical Medical Centre, University of Twente, Enschede.

Department of Pediatric Rheumatology, Division of Paediatrics, University Medical Center Utrecht, Wilhelmina Children's Hospital, Utrecht.

出版信息

Rheumatology (Oxford). 2023 Feb 23;62(SI2):SI170-SI180. doi: 10.1093/rheumatology/keac299.

Abstract

OBJECTIVE

To investigate medication prescription patterns among children with JIA, including duration, sequence and reasons for medication discontinuation.

METHODS

This study is a single-centre, retrospective analysis of prospective data from the electronic medical records of JIA patients receiving systemic therapy aged 0-18 years between 1 April 2011 and 31 March 2019. Patient characteristics (age, gender, JIA subtype) and medication prescriptions were extracted and analysed using descriptive statistics, Sankey diagrams and Kaplan-Meier survival methods.

RESULTS

Over a median of 4.2 years follow-up, the 20 different medicines analysed were prescribed as monotherapy (n = 15) or combination therapy (n = 48 unique combinations) among 236 patients. In non-systemic JIA, synthetic DMARDs were prescribed to almost all patients (99.5%), and always included MTX. In contrast, 43.9% of non-systemic JIA patients received a biologic DMARD (mostly adalimumab or etanercept), ranging from 30.9% for oligoarticular persistent ANA-positive JIA, to 90.9% for polyarticular RF-positive JIA. Among systemic JIA, 91.7% received a biologic DMARD (always including anakinra). When analysing medication prescriptions according to their class, 32.6% involved combination therapy. In 56.8% of patients, subsequent treatment lines were initiated after unsuccessful first-line treatment, resulting in 68 unique sequences. Remission was the most common reason for DMARD discontinuation (44.7%), followed by adverse events (28.9%) and ineffectiveness (22.1%).

CONCLUSION

This paper reveals the complexity of pharmacological treatment in JIA, as indicated by: the variety of mono- and combination therapies prescribed, substantial variation in medication prescriptions between subtypes, most patients receiving two or more treatment lines, and the large number of unique treatment sequences.

摘要

目的

研究幼年特发性关节炎(JIA)患儿的用药模式,包括用药持续时间、用药顺序和停药原因。

方法

本研究为单中心、回顾性分析,纳入 2011 年 4 月 1 日至 2019 年 3 月 31 日期间在我院接受全身治疗的 0-18 岁 JIA 患者的电子病历中的前瞻性数据。采用描述性统计、Sankey 图和 Kaplan-Meier 生存分析方法提取患者特征(年龄、性别、JIA 亚型)和用药方案。

结果

在中位数为 4.2 年的随访期间,在 236 例患者中,20 种不同的药物分别作为单药治疗(n=15)或联合治疗(n=48 种不同的联合治疗方案)。在非系统性 JIA 中,合成 DMARDs 几乎全部患者(99.5%),且均包括 MTX。相比之下,43.9%的非系统性 JIA 患者接受生物 DMARD(多为阿达木单抗或依那西普),范围从少关节型持续性抗核抗体阳性 JIA 的 30.9%到多关节型 RF 阳性 JIA 的 90.9%。在系统性 JIA 中,91.7%的患者接受生物 DMARD(始终包括阿那白滞素)。根据药物类别分析用药方案时,32.6%涉及联合治疗。在 56.8%的患者中,一线治疗失败后开始使用二线治疗方案,导致出现 68 种不同的治疗方案。DMARD 停药的最常见原因是缓解(44.7%),其次是不良事件(28.9%)和无效(22.1%)。

结论

本文揭示了 JIA 药物治疗的复杂性,具体表现在:处方的单药和联合治疗方案种类繁多,不同亚型之间药物治疗方案存在显著差异,大多数患者接受两种或更多种治疗方案,以及大量独特的治疗方案序列。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a5e0/9949706/bd02e84b149c/keac299f1.jpg

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