背景:系统性幼年特发性关节炎(sJIA)是一种严重的幼年关节炎,其特征是慢性关节炎症和发热等全身症状,皮疹,器官参与。抗IL-6受体单克隆抗体托珠单抗是一种有效的治疗方法。然而,一些患者仍有持续或反复发作的症状,从未报道过canakinumab在中国sJIA患者中的真实疗效.因此,本研究旨在使用真实世界数据评估canakinumab在中国sJIA患者中的疗效和安全性.
方法:我们对患有活动性sJIA的儿童进行了回顾性研究。临床特征,实验室数据,皮质类固醇剂量,和不良事件(AE)在基线和开始canakinumab治疗后4,8,12和24周收集.
结果:研究中包括7名女性和4名男性患者。所有患者以前都接受过托珠单抗治疗,并接受了卡纳单抗治疗12.4±3.4个月。值得注意的是,临床体征和症状以及实验室指标均有显著改善.4名接受皮质类固醇治疗的儿童成功停止了皮质类固醇治疗:1名在第4周,2名在第12周,1名在第24周。值得注意的是,关节压痛和肿胀的数量(P=0.0059)以及系统性幼年关节炎疾病活动评分(P<0.0001)显着减少。最常见的AE是感染,但没有患者出现严重的AE。随访期间无巨噬细胞活化综合征或死亡病例。
结论:在中国sJIA患者中发现Canakinumab可能是有效和安全的。canakinumab治疗未观察到新的AE。
BACKGROUND: 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.
METHODS: 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.
RESULTS: 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.
CONCLUSIONS: Canakinumab was found to be potentially efficacious and safe in Chinese patients with sJIA. No new AEs were observed with
canakinumab treatment.