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托珠单抗治疗静脉注射免疫球蛋白抵抗型川崎病的疗效与安全性病例系列:5例患者的回顾性分析

Case Series on the Efficacy and Safety of Tocilizumab in IVIG-Resistant Kawasaki Disease: A Retrospective Analysis of Five Patients.

作者信息

Ling Jiayun, Xie Fang, Zhou Qingfang, Ouyang Qian, Li Lin, Zhao Wei, Liu Xiaohui

机构信息

Department of Rheumatology and Immunology, Jiangxi Children's Hospital, Jiangxi Medical College, Nanchang, Jiangxi, People's Republic of China.

Pediatric Cardiology Treatment Center, Jiangxi Children's Hospital, Jiangxi Medical College, Nanchang, Jiangxi, People's Republic of China.

出版信息

J Inflamm Res. 2024 Dec 11;17:10991-10998. doi: 10.2147/JIR.S479879. eCollection 2024.

Abstract

BACKGROUND

Kawasaki disease (KD) is a leading cause of acquired heart disease in children in developed countries. While intravenous immunoglobulin (IVIG) is the standard treatment, 10-20% of patients do not respond to IVIG. Given the significant role of interleukin-6 (IL-6) in KD pathophysiology, targeting IL-6 may offer a therapeutic option for IVIG-resistant KD.

OBJECTIVE

This study aims to evaluate the efficacy and safety of tocilizumab (an IL-6 receptor antagonist) in the treatment of IVIG-resistant KD through a retrospective analysis of clinical data.

METHODS

Clinical data from five KD patients treated with tocilizumab (TCZ) at Jiangxi Children's Hospital from January 2023 to February 2024 were retrospectively analyzed. All patients met the 2017 American Heart Association diagnostic criteria for KD and received a single dose of TCZ. Data on clinical manifestations, laboratory indicators, and coronary artery changes before and after TCZ treatment were collected.

RESULTS

Four of the five patients had a KOBAYASHI score >4, and initial IVIG and glucocorticoid treatments were ineffective. Following TCZ administration, body temperature normalized within 12 hours in three patients, and inflammatory markers, including C-reactive protein and interleukin-6, returned to normal levels. Coronary artery dilation was observed in 75% of patients pre-TCZ; post-TCZ, coronary arteries normalized in three patients, while one patient experienced progressive right coronary artery dilation. No adverse events were reported following TCZ administration.

CONCLUSION

Tocilizumab effectively controls systemic inflammation and improves coronary artery outcomes in IVIG-resistant KD patients, with no observed adverse events. Larger studies are necessary to confirm these findings and further investigate long-term effects on coronary artery health.

摘要

背景

川崎病(KD)是发达国家儿童获得性心脏病的主要病因。虽然静脉注射免疫球蛋白(IVIG)是标准治疗方法,但10%-20%的患者对IVIG无反应。鉴于白细胞介素-6(IL-6)在KD病理生理学中的重要作用,靶向IL-6可能为IVIG抵抗性KD提供一种治疗选择。

目的

本研究旨在通过对临床数据的回顾性分析,评估托珠单抗(一种IL-6受体拮抗剂)治疗IVIG抵抗性KD的疗效和安全性。

方法

回顾性分析2023年1月至2024年2月在江西省儿童医院接受托珠单抗(TCZ)治疗的5例KD患者的临床资料。所有患者均符合2017年美国心脏协会KD诊断标准,并接受单剂量TCZ治疗。收集TCZ治疗前后的临床表现、实验室指标和冠状动脉变化数据。

结果

5例患者中有4例小林评分>4,初始IVIG和糖皮质激素治疗无效。给予TCZ后,3例患者体温在12小时内恢复正常,包括C反应蛋白和白细胞介素-6在内的炎症标志物恢复到正常水平。75%的患者在TCZ治疗前观察到冠状动脉扩张;TCZ治疗后,3例患者的冠状动脉恢复正常,而1例患者出现右冠状动脉进行性扩张。给予TCZ后未报告不良事件。

结论

托珠单抗可有效控制IVIG抵抗性KD患者的全身炎症并改善冠状动脉结局,未观察到不良事件。需要更大规模的研究来证实这些发现,并进一步研究其对冠状动脉健康的长期影响。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/288e/11646367/5af1739c6c1a/JIR-17-10991-g0001.jpg

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