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卡那奴单抗治疗冷吡啉相关周期性综合征。

Canakinumab for treatment of cryopyrin-associated periodic syndrome.

机构信息

Department of Rheumatology and Clinical Immunology, Charité - Universitätsmedizin Berlin, Charitéplatz 1, D-10117 Berlin, Germany.

出版信息

Expert Opin Biol Ther. 2010 Nov;10(11):1631-6. doi: 10.1517/14712598.2010.530653.

DOI:10.1517/14712598.2010.530653
PMID:20955115
Abstract

IMPORTANCE OF THE FIELD

Autoinflammatory syndromes such as cryopyrin-associated periodic syndromes (CAPS) place a heavy burden on affected individuals as well as on their families due to significant morbidity and increased mortality. The inflammatory response in CAPS is caused by an overwhelming activation of the pro-inflammatory cytokine IL-1, which was identified as a promising treatment target.

AREAS COVERED IN THIS REVIEW

This article focuses on the pathogenic background and different clinical manifestations in CAPS. Furthermore, the development program and characteristics of canakinumab, a recently approved fully human anti-IL-1β mAb for the treatment of CAPS, are described and compared to other available IL-1 blocking agents.

WHAT THE READER WILL GAIN

Canakinumab targets selectively human IL-1ß with high affinity and prevents the cytokine from interaction to its receptor and, thus, effectively blocks the inflammatory response in CAPS. In all studies performed, canakinumab showed a rapid improvement of symptoms of CAPS and a complete clinical response was achieved in most patients. Inflammatory markers such as C-reactive protein and serum amyloid-A protein were reduced to normal levels within few days. In comparison to other IL-1 blockers, canakinumab provides a longer plasma half-life and less injection site reactions.

TAKE HOME MESSAGE

Canakinumab offers the possibility of permanent disease control, almost symptom-free life, and hopefully less long-term morbidity and mortality in patients with CAPS.

摘要

重要性领域

自炎症性疾病,如 Cryopyrin 相关周期性综合征(CAPS),由于发病率高和死亡率增加,给患者及其家属带来了沉重的负担。CAPS 中的炎症反应是由过度激活促炎细胞因子 IL-1 引起的,IL-1 已被确定为一种有前途的治疗靶点。

本文综述了 CAPS 的发病机制背景和不同临床表现。此外,还描述了最近批准的用于治疗 CAPS 的全人源抗 IL-1β mAb 卡那单抗的开发项目和特点,并与其他可用的 IL-1 阻断剂进行了比较。

读者将获得什么

卡那单抗以高亲和力选择性靶向人 IL-1β,阻止细胞因子与其受体相互作用,从而有效阻断 CAPS 中的炎症反应。在所有进行的研究中,卡那单抗迅速改善了 CAPS 的症状,大多数患者达到了完全临床缓解。在几天内,炎症标志物如 C 反应蛋白和血清淀粉样蛋白 A 蛋白降至正常水平。与其他 IL-1 阻滞剂相比,卡那单抗提供了更长的血浆半衰期和更少的注射部位反应。

带回家的信息

卡那单抗为 CAPS 患者提供了永久性疾病控制、几乎无症状的生活以及降低长期发病率和死亡率的可能性。

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Canakinumab for treatment of cryopyrin-associated periodic syndrome.卡那奴单抗治疗冷吡啉相关周期性综合征。
Expert Opin Biol Ther. 2010 Nov;10(11):1631-6. doi: 10.1517/14712598.2010.530653.
2
Canakinumab: a human anti-IL-1β monoclonal antibody for the treatment of cryopyrin-associated periodic syndromes.卡那奴单抗:一种人源抗 IL-1β 单克隆抗体,用于治疗 Cryopyrin 相关周期性综合征。
Expert Rev Clin Immunol. 2010 Nov;6(6):831-41. doi: 10.1586/eci.10.66.
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Real-life effectiveness of canakinumab in cryopyrin-associated periodic syndrome.结晶相关周期性综合征中卡那奴单抗的实际疗效。
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Canakinumab (ACZ885, a fully human IgG1 anti-IL-1β mAb) induces sustained remission in pediatric patients with cryopyrin-associated periodic syndrome (CAPS).卡那奴单抗(ACZ885,一种完全人源 IgG1 抗 IL-1β mAb)可诱导 Cryopyrin 相关周期性综合征(CAPS)儿科患者的持续缓解。
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Two-year results from an open-label, multicentre, phase III study evaluating the safety and efficacy of canakinumab in patients with cryopyrin-associated periodic syndrome across different severity phenotypes.评估卡那奴单抗在不同严重表型的 Cryopyrin 相关周期性综合征患者中的安全性和疗效的开放性、多中心、III 期研究的两年结果。
Ann Rheum Dis. 2011 Dec;70(12):2095-102. doi: 10.1136/ard.2011.152728. Epub 2011 Aug 21.
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Pharmacological treatment options for cryopyrin-associated periodic syndromes.冷吡啉相关周期性综合征的药物治疗选择
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Canakinumab for the treatment of cryopyrin-associated periodic syndromes.卡那单抗用于治疗冷吡啉相关周期性综合征。
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Efficacy and safety of canakinumab in cryopyrin-associated periodic syndromes: results from a Spanish cohort.卡那单抗治疗冷吡啉相关周期性综合征的疗效和安全性:来自西班牙队列的结果
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Efficacy and safety of canakinumab therapy in paediatric patients with cryopyrin-associated periodic syndrome: a single-centre, real-world experience.卡那奴单抗治疗 cryopyrin 相关周期性综合征儿科患者的疗效和安全性:单中心真实世界经验。
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