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托珠单抗单药治疗显微镜下多血管炎的无皮质类固醇治疗:一项单臂、单中心临床试验。

Corticosteroid-free treatment of tocilizumab monotherapy for microscopic polyangiitis: a single-arm, single-center, clinical trial.

机构信息

a Department of Rheumatology and Clinical Immunology , Saitama Medical Center, Saitama Medical University , Kawagoe , Japan.

出版信息

Mod Rheumatol. 2016 Nov;26(6):900-907. doi: 10.3109/14397595.2016.1160968. Epub 2016 Apr 21.

Abstract

OBJECTIVES

To assess the efficacy of tocilizumab (TCZ) monotherapy for the remission induction of microscopic polyangiitis (MPA) in a prospective single-arm, single-center, cohort, pilot study.

METHODS

Eligible patients were aged between 20 and 80 years and were newly diagnosed with MPA according to Watts' classification algorithm. Seven patients received 8 mg/kg of intravenous TCZ fortnightly for the first 2 months (5 courses), and monthly for the next 10 months (10 courses). One year after TCZ monotherapy, the patients were followed-up without any treatment. The protocol did not permit the use corticosteroids or any other immunosuppressants. Complete remission (CR) was defined as the Birmingham Vasculitis Activity Score of 0 at two consecutive visits made at least a month apart.

RESULTS

CR was achieved in two of six patients (33.3%) at 6 months and three patients (50.0%) at 12 months. Two patients were withdrawn: one because of inefficacy at 6 weeks and the other because of flare at 6 months. One patient voluntarily withdrew after CR at 3 months. Four patients (66.7%) could be kept drug-free after 1 year of TCZ without relapse for 6-15 months at the last visit.

CONCLUSION

TCZ monotherapy may be an alternative treatment strategy in some patients with MPA.

摘要

目的

评估托珠单抗(TCZ)单药治疗在一项前瞻性单臂、单中心、队列、初步研究中对显微镜下多血管炎(MPA)缓解诱导的疗效。

方法

符合条件的患者年龄在 20 至 80 岁之间,根据 Watts 分类算法新诊断为 MPA。7 名患者在前 2 个月每 2 周接受 8mg/kg 的静脉 TCZ(5 个疗程),然后每月接受 10 个月(10 个疗程)。在 TCZ 单药治疗 1 年后,患者无需任何治疗即可进行随访。方案不允许使用皮质类固醇或任何其他免疫抑制剂。完全缓解(CR)定义为两次连续就诊,至少相隔一个月,伯明翰血管炎活动评分均为 0。

结果

6 个月时 6 例患者中有 2 例(33.3%)达到 CR,12 个月时 3 例(50.0%)达到 CR。2 例患者退出:1 例在 6 周时无效,另 1 例在 6 个月时复发。1 例患者在 3 个月时达到 CR 后自愿退出。在 TCZ 治疗 1 年后,4 例患者(66.7%)无需继续使用药物,无复发,在最后一次随访时 6-15 个月未复发。

结论

TCZ 单药治疗可能是某些 MPA 患者的一种替代治疗策略。

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