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现实环境中生物疗法对非系统性幼年特发性关节炎患儿的文献分析及意义

Literature analysis and implication of biologic therapy for children with non-systemic juvenile idiopathic arthritis in real-world settings.

作者信息

Fung Albert, Yue Xiaomeng, Wigle Patricia R, Guo Jeff J

机构信息

Division of Pharmacy Practice and Administrative Sciences, James L. Winkle College of Pharmacy, University of Cincinnati Academic Health Center, Cincinnati, Ohio, USA.

出版信息

Intractable Rare Dis Res. 2025 Aug 31;14(3):162-174. doi: 10.5582/irdr.2025.01035.

Abstract

Juvenile idiopathic arthritis (JIA) is the most common rheumatological disease in children. Besides the more severe systemic form, non-systemic JIA is divided into 5 different subgroups. Polyarticular JIA (polyJIA), particularly rheumatoid factor (RF)-positive, which is defined as the disease involving five or more joints in the first 6 months of disease, has the worst prognosis. Biologic disease-modifying antirheumatic drugs (bDMARDs), particularly tumor necrosis factor inhibitors (TNFi), are the backbone of JIA treatment regimens. This research analyzed the published articles for: ) optimal sequence, timing and outcomes; ) comparative effectiveness of various bDMARDs; and ) safety concerns for use of bDMARDs. For patients with polyJIA, early effective treatment with bDMARDs is associated with drug-free remission, lower disease activity, better disease control and outcomes. Adalimumab, etanercept and tocilizumab have comparable effectiveness for treating polyJIA, and these drugs are also well-tolerated. JIA patients had a higher rate of hospitalized/serious infection and malignancy compared to the general population. The use of TNFi did not seem to significantly increase this risk further when compared to using methotrexate. Patients treated with IL-1 inhibitors or IL-6 inhibitors reported significantly more serious infections, compared with patients treated with TNFi. Clinicians and patients should consider potential risk in light of benefits of bDMARDs. The reimbursement policy and pricing issue of bDMARDs are out of the scope of the present literature analysis. The current review may help inform shared decision-making discussions between families and physicians as they weigh the risks and benefits of various treatment approaches for children with JIA.

摘要

幼年特发性关节炎(JIA)是儿童中最常见的风湿性疾病。除了更严重的全身型外,非全身型JIA可分为5个不同的亚组。多关节型JIA(polyJIA),尤其是类风湿因子(RF)阳性的类型,被定义为在疾病的前6个月累及5个或更多关节,其预后最差。生物性改善病情抗风湿药物(bDMARDs),尤其是肿瘤坏死因子抑制剂(TNFi),是JIA治疗方案的核心。本研究分析了已发表的文章,以探讨:(1)最佳顺序、时机和结果;(2)各种bDMARDs的比较疗效;以及(3)使用bDMARDs的安全性问题。对于多关节型JIA患者,早期使用bDMARDs进行有效治疗与无药物缓解、较低的疾病活动度、更好的疾病控制和结果相关。阿达木单抗、依那西普和托珠单抗在治疗多关节型JIA方面具有相当的疗效,并且这些药物耐受性良好。与普通人群相比,JIA患者住院/严重感染和恶性肿瘤的发生率更高。与使用甲氨蝶呤相比,使用TNFi似乎并未进一步显著增加这种风险。与使用TNFi治疗的患者相比,使用IL-1抑制剂或IL-6抑制剂治疗的患者报告的严重感染明显更多。临床医生和患者应根据bDMARDs的益处考虑潜在风险。bDMARDs的报销政策和定价问题不在本文献分析范围内。本综述可能有助于为家庭和医生之间的共同决策讨论提供信息,因为他们在权衡JIA患儿各种治疗方法的风险和益处。

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本文引用的文献

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