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卡那单抗治疗全身型幼年特发性关节炎的疗效和安全性:中国首项临床经验

Efficacy and safety of canakinumab in systemic juvenile idiopathic arthritis, the first Chinese experience.

机构信息

Department of Rheumatology and Immunology, Children's Hospital of Nanjing Medical University, No. 72 Guangzhou Road, Nanjing, Jiangsu Province, 210008, China.

出版信息

Pediatr Rheumatol Online J. 2024 Mar 19;22(1):38. doi: 10.1186/s12969-024-00974-4.

Abstract

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.

摘要

背景

全身型幼年特发性关节炎(sJIA)是一种严重的幼年型关节炎,其特征为慢性关节炎症和全身症状,如发热、皮疹和器官受累。抗白细胞介素 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复发的症状,卡那单抗在中国 sJIA 患者中的真实世界疗效尚未报道。因此,本研究旨在使用真实世界数据评估卡那单抗在中国 sJIA 患者中的疗效和安全性。

方法

我们对活动性 sJIA 患儿进行了回顾性研究。在开始使用卡那单抗治疗前和治疗后第 4、8、12 和 24 周时,收集了临床特征、实验室数据、皮质类固醇剂量和不良事件(AE)。

结果

本研究纳入 7 例女性和 4 例男性患者。所有患者之前均接受过托珠单抗治疗,并接受卡那单抗治疗 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显著改善。4 例接受皮质类固醇治疗的患儿成功停用皮质类固醇:1 例在第 4 周,2 例在第 12 周,1 例在第 24 周。值得注意的是,压痛和肿胀关节数(P=0.0059)以及全身幼年特发性关节炎疾病活动评分(P<0.0001)均显著降低。最常见的 AE 是感染,但无患者发生严重 AE。在随访期间,未报告巨噬细胞活化综合征或死亡病例。

结论

卡那单抗在中国 sJIA 患者中具有潜在疗效和安全性。卡那单抗治疗未观察到新的 AE。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/160e/10949691/f41e1a50769c/12969_2024_974_Fig1_HTML.jpg

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