PDF(Sci-hub)
Abstract：
Several therapies are used for the treatment of rareautoinflammatory conditions like cryopyrin-associated periodic fever syndromes (CAPS), hyperimmunoglobulin Dsyndrome (HIDS)/mevalonate kinase deficiency (MKD) and tumour necrosis factor receptor-associated periodic syndrome (TRAPS). However, reviews reporting on treatment outcomes of these therapies are lacking.
A systematic literature review was conducted using Embase, MEDLINE, MEDLINE-In Process and Cochrane databases to identify the randomised/non-randomised controlled trials (RCTs/non-RCTs) and real-world observational studies of CAPS, HIDS/MKD and TRAPS published as full-texts (January 2000-September 2017) or conference abstracts (January 2014-September 2017). Studies with data for ≥1 biologic were included. Studies with <5 patients were excluded.
Of the 3 342 retrieved publications, 72 studies were included (CAPS, n=43; HIDS/MKD, n=9; TRAPS, n=7; studies with ≥2 cohorts, n=13). Most studies were full-text (n=56), published after 2010 (n=56) and real-world observational studies (n=58). Among included studies, four were RCTs (canakinumab, n=2 (CAPS, n=1; HIDS/MKD and TRAPS, n=1); rilonacept, n=1 (in CAPS); simvastatin, n=1 (in HIDS/MKD)). Canakinumab and anakinra were the most commonly used therapies for CAPS and HIDS/MKD, whereas etanercept, canakinumab and anakinra were the most common for TRAPS. The available evidence suggested the efficacy or effectiveness of canakinumab and anakinra in CAPS, HIDS/MKD and TRAPS, and of etanercept in TRAPS; asingle RCT demonstrated the efficacy of rilonacept in CAPS.
Canakinumab, anakinra, etanercept and rilonacept were reported to be well tolerated; however, injection-site reactions were observed frequently with anakinra, rilonacept and etanercept. Data on the use of tocilizumab, infliximab and adalimumab in these conditions were limited; thus, further research is warranted.
A systematic literature review was conducted using Embase, MEDLINE, MEDLINE-In Process and Cochrane databases to identify the randomised/non-randomised controlled trials (RCTs/non-RCTs) and real-world observational studies of CAPS, HIDS/MKD and TRAPS published as full-texts (January 2000-September 2017) or conference abstracts (January 2014-September 2017). Studies with data for ≥1 biologic were included. Studies with <5 patients were excluded.
Of the 3 342 retrieved publications, 72 studies were included (CAPS, n=43; HIDS/MKD, n=9; TRAPS, n=7; studies with ≥2 cohorts, n=13). Most studies were full-text (n=56), published after 2010 (n=56) and real-world observational studies (n=58). Among included studies, four were RCTs (canakinumab, n=2 (CAPS, n=1; HIDS/MKD and TRAPS, n=1); rilonacept, n=1 (in CAPS); simvastatin, n=1 (in HIDS/MKD)). Canakinumab and anakinra were the most commonly used therapies for CAPS and HIDS/MKD, whereas etanercept, canakinumab and anakinra were the most common for TRAPS. The available evidence suggested the efficacy or effectiveness of canakinumab and anakinra in CAPS, HIDS/MKD and TRAPS, and of etanercept in TRAPS; asingle RCT demonstrated the efficacy of rilonacept in CAPS.
Canakinumab, anakinra, etanercept and rilonacept were reported to be well tolerated; however, injection-site reactions were observed frequently with anakinra, rilonacept and etanercept. Data on the use of tocilizumab, infliximab and adalimumab in these conditions were limited; thus, further research is warranted.
摘要：
几种疗法用于治疗罕见的自身炎症，如冷冻比林相关的周期性发热综合征（CAPS），高免疫球蛋白D综合征（HIDS）/甲羟戊酸激酶缺乏症（MKD）和肿瘤坏死因子受体相关周期性综合征（TRAPS）。然而,缺乏关于这些疗法治疗结果的综述报告.
使用Embase进行了系统的文献综述，MEDLINE,MEDLINE-InProcess和Cochrane数据库，用于确定CAPS的随机/非随机对照试验（RCT/非RCT）和真实世界观察性研究，HIDS/MKD和TRAPS以全文（2000年1月至2017年9月）或会议摘要（2014年1月至2017年9月）发布。包括≥1个生物学数据的研究。排除<5名患者的研究。
在检索到的3342份出版物中,包括72项研究(CAPS，n=43；HIDS/MKD，n=9；陷阱，n=7；具有≥2个队列的研究，n=13)。大多数研究是全文(n=56)，2010年后发表的(n=56)和真实世界的观察研究(n=58)。在纳入的研究中，四个是随机对照试验(canakinumab，n=2(CAPS，n=1；HIDS/MKD和TRAPS，n=1)；rilonacept，n=1(CAPS)；辛伐他汀，n=1(在HIDS/MKD中)。Canakinumab和anakinra是CAPS和HIDS/MKD最常用的疗法，而依那西普，canakinumab和anakinra是TRAPS最常见的.现有证据表明canakinumab和anakinra在CAPS中的疗效或有效性，HIDS/MKD和陷阱，和etanercept在TRAPS中的应用；asingleRCT证明了利洛纳康在CAPS中的疗效。
Canakinumab,anakinra,据报道，依那西普和利洛纳西普的耐受性良好；然而，经常观察到Anakinra的注射部位反应，rilonacept和etanercept。使用托珠单抗的数据，英夫利昔单抗和阿达木单抗在这些情况下是有限的；因此，需要进一步的研究。
使用Embase进行了系统的文献综述，MEDLINE,MEDLINE-InProcess和Cochrane数据库，用于确定CAPS的随机/非随机对照试验（RCT/非RCT）和真实世界观察性研究，HIDS/MKD和TRAPS以全文（2000年1月至2017年9月）或会议摘要（2014年1月至2017年9月）发布。包括≥1个生物学数据的研究。排除<5名患者的研究。
在检索到的3342份出版物中,包括72项研究(CAPS，n=43；HIDS/MKD，n=9；陷阱，n=7；具有≥2个队列的研究，n=13)。大多数研究是全文(n=56)，2010年后发表的(n=56)和真实世界的观察研究(n=58)。在纳入的研究中，四个是随机对照试验(canakinumab，n=2(CAPS，n=1；HIDS/MKD和TRAPS，n=1)；rilonacept，n=1(CAPS)；辛伐他汀，n=1(在HIDS/MKD中)。Canakinumab和anakinra是CAPS和HIDS/MKD最常用的疗法，而依那西普，canakinumab和anakinra是TRAPS最常见的.现有证据表明canakinumab和anakinra在CAPS中的疗效或有效性，HIDS/MKD和陷阱，和etanercept在TRAPS中的应用；asingleRCT证明了利洛纳康在CAPS中的疗效。
Canakinumab,anakinra,据报道，依那西普和利洛纳西普的耐受性良好；然而，经常观察到Anakinra的注射部位反应，rilonacept和etanercept。使用托珠单抗的数据，英夫利昔单抗和阿达木单抗在这些情况下是有限的；因此，需要进一步的研究。
