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接受生物性改善病情抗风湿药物治疗的幼年特发性关节炎患儿的治疗结果:来自印度的一项回顾性单中心经验

Outcomes of children with juvenile idiopathic arthritis receiving biological disease-modifying anti-rheumatic drugs: a retrospective single-centre experience from India.

作者信息

Kaur Pavneet, Kizhakkeveettil Saheer Farheen, Rama Krishna J Bala Siva, Sreeshanth Banoth, Kavil Peedika Ayisha, Thrisha Reddy Bareddy Sai, Upadhyay Ashish Datt, Lodha Rakesh, Kabra Sushil Kumar, Bagri Narendra

机构信息

Department of Paediatrics, All India Institute of Medical Sciences, New Delhi, India.

All India Institute of Medical Sciences, New Delhi, India.

出版信息

Lancet Reg Health Southeast Asia. 2025 Jun 5;38:100612. doi: 10.1016/j.lansea.2025.100612. eCollection 2025 Jul.

Abstract

BACKGROUND

We aimed to study the outcomes (remission, flare and adverse events) of biological disease-modifying anti-rheumatic drugs (bDMARD) in children with JIA from a low-middle-income country setting, and explore the factors associated with these outcomes.

METHODS

The Pediatric Rheumatology Clinic bDMARD register (2009 to August 2024) was screened to enrol children with JIA and at least 3 months follow-up whilst on bDMARDs. Participant characteristics and clinical responses were collected in a pre-designed proforma to evaluate the primary objective i.e., studying outcomes among children with JIA on bDMARDs. The secondary objective was to study factors associated with time-to-remission (TTR) and flare-after-stopping-bDMARDs.

FINDINGS

One-hundred-fifteen children (59.1% boys) with 168 patient-years of bDMARD use were enrolled for this single-centre study. Enthesitis-related arthritis was the commonest subtype of JIA (n = 44, 38.3%). The most commonly used bDMARD was adalimumab (n = 43, 37.3%). The median (IQR) delay to initiation of bDMARD from the perceived need was 2 (0-6) months, primarily due to financial impediments (n = 81, 70.4%). Fifteen (13%) children screened positive for tuberculosis infection. One hundred ten (95.6%) children achieved remission on bDMARD, after a median (IQR) of 7.5 (4-12) weeks. Macrophage activation syndrome at initiation was significantly associated (HR 3.6 (1.3-10.0), p = 0.03) with a longer time-to-remission. bDMARDs were stopped in n = 68/115 (59.1%) after a median (IQR) 15 (9.6-26.5) months, of whom n = 33/68 (48.5%) flared at 6 (3.5-12) months of follow-up. A longer time-to-remission (OR 1.12 (1.02-1.23), p = 0.01) was significantly associated with flare after stopping bDMARDs. Forty-two (36.5%) patients experienced adverse events. The most striking adverse events were serious infections requiring hospitalisation (n = 13, 11.3%) and tuberculosis (n = 4, 3.5%). All children who developed tuberculosis were on TNFi (Adalimumab).

INTERPRETATION

Though children on bDMARDs showed comparable remission rates, we noted a higher frequency of serious infections and tuberculosis, compared to the experience described from high-income countries. These observations highlight the need for further surveillance of outcomes of bDMARD use among children with JIA in an LMIC setting.

FUNDING

There has been no financial support for this work.

摘要

背景

我们旨在研究中低收入国家环境下幼年特发性关节炎(JIA)儿童使用生物改善病情抗风湿药物(bDMARD)的结局(缓解、病情复发和不良事件),并探索与这些结局相关的因素。

方法

筛查儿科风湿病诊所bDMARD登记册(2009年至2024年8月),纳入JIA儿童且在使用bDMARD期间至少随访3个月。在预先设计的表格中收集参与者特征和临床反应,以评估主要目标,即研究使用bDMARD的JIA儿童的结局。次要目标是研究与缓解时间(TTR)和停用bDMARD后病情复发相关的因素。

结果

115名儿童(59.1%为男孩),使用bDMARD的患者年数为168年,纳入了这项单中心研究。附着点炎相关关节炎是JIA最常见的亚型(n = 44,38.3%)。最常用的bDMARD是阿达木单抗(n = 43,37.3%)。从意识到需要开始使用bDMARD的中位(IQR)延迟时间为2(0 - 6)个月,主要原因是经济障碍(n = 81,70.4%)。15名(13%)儿童结核感染筛查呈阳性。110名(95.6%)儿童在使用bDMARD后达到缓解,中位(IQR)时间为7.5(4 - 12)周。起始时巨噬细胞活化综合征与缓解时间延长显著相关(HR 3.6(1.3 - 10.0),p = 0.03)。68/115(59.1%)名儿童在中位(IQR)15(9.6 - 26.5)个月后停用bDMARD,其中33/68(48.5%)名儿童在随访6(3.5 - 12)个月时病情复发。缓解时间延长(OR 1.12(1.02 - 1.23),p = 0.01)与停用bDMARD后病情复发显著相关。42名(36.5%)患者发生不良事件。最突出的不良事件是需要住院治疗的严重感染(n = 13,11.3%)和结核病(n = 4,3.5%)。所有发生结核病的儿童均使用肿瘤坏死因子抑制剂(TNFi,阿达木单抗)。

解读

尽管使用bDMARD的儿童缓解率相当,但与高收入国家描述的情况相比,我们注意到严重感染和结核病的发生率更高。这些观察结果凸显了在中低收入国家环境下,需要进一步监测JIA儿童使用bDMARD的结局。

资金

这项工作没有获得资金支持。

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