卡那单抗治疗系统性幼年特发性关节炎的疗效和安全性--中国首次应用经验

IF 2.8 3区 医学 Q1 PEDIATRICS
Lingzhi Qiu, Le Ma, Yifan Xie, Jing Jin, Yuting Pan, Shumin Li, Zhidan Fan, Haiguo Yu
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引用次数: 0

摘要

全身性幼年特发性关节炎(sJIA)是一种严重的幼年关节炎,其特点是慢性关节炎症和全身症状,如发热、皮疹和器官受累。抗IL-6受体单克隆抗体托西珠单抗是一种有效的治疗方法。然而,部分患者仍会出现持续或反复发作的症状,而卡那珠单抗在中国sJIA患者中的实际疗效尚未见报道。因此,本研究旨在利用真实世界的数据评估卡那珠单抗在中国sJIA患者中的疗效和安全性。我们对活动性sJIA患儿进行了一项回顾性研究。我们在基线和开始卡那单抗治疗后的 4、8、12 和 24 周收集了临床特征、实验室数据、皮质类固醇用量和不良事件(AEs)。研究共纳入了七名女性患者和四名男性患者。所有患者之前都接受过托西珠单抗治疗,卡那单抗的疗程为(12.4 ± 3.4)个月。值得注意的是,临床症状和体征以及实验室指标均有明显改善。四名接受皮质类固醇治疗的患儿成功地停止了皮质类固醇治疗:一名患儿在第4周停止治疗,两名患儿在第12周停止治疗,一名患儿在第24周停止治疗。值得注意的是,压痛和肿胀关节的数量(P = 0.0059)以及全身性幼年关节炎疾病活动度评分(P < 0.0001)均明显减少。最常见的不良反应是感染,但没有患者出现严重不良反应。随访期间没有出现巨噬细胞活化综合征或死亡病例。卡纳库单抗对中国sJIA患者具有潜在疗效和安全性。卡纳库单抗治疗未发现新的AEs。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Efficacy and safety of canakinumab in systemic juvenile idiopathic arthritis, the first Chinese experience
Systemic juvenile idiopathic arthritis (sJIA) is a severe form of juvenile arthritis that is characterized by chronic joint inflammation and systemic symptoms such as fever, rash, and organ involvement. Anti-IL-6 receptor monoclonal antibody tocilizumab is an effective treatment. However, some patients still experience persisting or recurrent symptoms and the real-world effectiveness of canakinumab in Chinese patients with sJIA has never been reported. Therefore, this study aimed to assess the efficacy and safety of canakinumab in Chinese patients with sJIA using real-world data. We conducted a retrospective study on children with active sJIA. Clinical features, laboratory data, corticosteroid dosage, and adverse events (AEs) were collected at baseline and at 4, 8, 12, and 24 weeks after initiating canakinumab treatment. Seven female and four male patients were included in the study. All patients had previously been treated with tocilizumab and were administered canakinumab for 12.4 ± 3.4 months. Notably, significant improvements were observed in both clinical signs and symptoms as well as laboratory indicators. Four children under corticosteroid treatment were able to successfully discontinue their corticosteroid therapy: one at week 4, two at week 12, and one at week 24. Notably, there was a significant reduction in the number of tender and swollen joints (P = 0.0059) as well as the systemic juvenile arthritis disease activity score (P < 0.0001). The most common AE was infection, but no patients experienced serious AEs. No cases of macrophage activation syndrome or death were reported during the follow-up period. Canakinumab was found to be potentially efficacious and safe in Chinese patients with sJIA. No new AEs were observed with canakinumab treatment.
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来源期刊
Pediatric Rheumatology
Pediatric Rheumatology PEDIATRICS-RHEUMATOLOGY
CiteScore
4.10
自引率
8.00%
发文量
95
审稿时长
>12 weeks
期刊介绍: Pediatric Rheumatology is an open access, peer-reviewed, online journal encompassing all aspects of clinical and basic research related to pediatric rheumatology and allied subjects. The journal’s scope of diseases and syndromes include musculoskeletal pain syndromes, rheumatic fever and post-streptococcal syndromes, juvenile idiopathic arthritis, systemic lupus erythematosus, juvenile dermatomyositis, local and systemic scleroderma, Kawasaki disease, Henoch-Schonlein purpura and other vasculitides, sarcoidosis, inherited musculoskeletal syndromes, autoinflammatory syndromes, and others.
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