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依那西普治疗肿瘤坏死因子受体相关周期性综合征的疗效:一项前瞻性、开放标签、剂量递增研究。

Efficacy of etanercept in the tumor necrosis factor receptor-associated periodic syndrome: a prospective, open-label, dose-escalation study.

作者信息

Bulua Ariel C, Mogul Douglas B, Aksentijevich Ivona, Singh Harjot, He David Y, Muenz Larry R, Ward Michael M, Yarboro Cheryl H, Kastner Daniel L, Siegel Richard M, Hull Keith M

机构信息

National Institute of Arthritis and Musculoskeletal and Skin Diseases, NIH, Bethesda, MD 20892, USA.

出版信息

Arthritis Rheum. 2012 Mar;64(3):908-13. doi: 10.1002/art.33416.

DOI:10.1002/art.33416
PMID:22006113
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3882089/
Abstract

OBJECTIVE

To investigate the efficacy of etanercept in improving the symptoms and underlying inflammation in patients with tumor necrosis factor receptor-associated periodic syndrome (TRAPS).

METHODS

Fifteen patients with TRAPS were enrolled in a prospective, open-label, dose-escalation study. Patients recorded attacks, symptom severity, and use of ancillary medications in a daily diary. Blood samples were collected during each period and measured for levels of acute-phase reactants. Between 7 years and 9 years after the conclusion of the initial study, patients completed a followup survey and were evaluated to determine the long-term outcome of etanercept treatment.

RESULTS

Etanercept treatment significantly attenuated the total symptom score and reduced the frequency of symptoms. Etanercept also reduced levels of acute-phase reactants, particularly during asymptomatic periods. During a 10-year followup period, patients continued to receive etanercept for a median of 3.3 years, with a number of patients switching to anti-interleukin-1β receptor therapy or not receiving biologic agents, most frequently citing injection site reactions and lack of efficacy as reasons for discontinuation. However, patients continuing to receive etanercept had reduced symptoms at followup.

CONCLUSION

Etanercept reduces symptoms and serum levels of inflammatory markers of TRAPS in a dose-dependent manner, but does not completely normalize symptoms or acute-phase reactant levels. Although long-term adherence to etanercept is poor, continuing to receive etanercept may provide continued symptomatic benefit.

摘要

目的

探讨依那西普对肿瘤坏死因子受体相关周期性综合征(TRAPS)患者症状及潜在炎症的改善效果。

方法

15例TRAPS患者纳入一项前瞻性、开放标签、剂量递增研究。患者通过每日日记记录发作情况、症状严重程度及辅助药物使用情况。在每个阶段采集血样,检测急性期反应物水平。在初始研究结束7至9年后,患者完成随访调查,评估依那西普治疗的长期结果。

结果

依那西普治疗显著降低了总症状评分,减少了症状发作频率。依那西普还降低了急性期反应物水平,尤其是在无症状期。在10年随访期内,患者持续接受依那西普治疗的中位时间为3.3年,一些患者转而接受抗白细胞介素-1β受体治疗或未接受生物制剂治疗,最常提及的停药原因是注射部位反应和缺乏疗效。然而,继续接受依那西普治疗的患者在随访时症状减轻。

结论

依那西普以剂量依赖方式减轻TRAPS患者的症状及炎症标志物血清水平,但不能使症状或急性期反应物水平完全恢复正常。尽管依那西普的长期依从性较差,但继续接受依那西普治疗可能会持续带来症状改善。

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