Efficacy and safety of treatments in familial Mediterranean fever and its complications: a systematic review informing the EULAR/PReS recommendations for familial Mediterranean fever.

Methods

This was a systematic review(SR) of studies testing pharmacological treatments in FMF patients, including targets, dosing, tapering, and uses in AA amyloidosis.

这是一项系统评价(SR)，研究了在FMF患者中测试药物治疗的目标、剂量、减量以及在AA淀粉样变性中的应用。

MEDLINE, Embase, and the Cochrane Library were searched, focusing on studies published after October 1, 2014.

在MEDLINE、Embase和Cochrane图书馆进行了检索，重点关注2014年10月1日之后发表的研究。

The Cochrane Risk of Bias tool and the Newcastle-Ottawa Scale were used to assess the risk of bias in the included randomised controlled trials (RCTs) and observational studies, respectively.

Cochrane风险偏倚工具和纽卡斯尔-渥太华量表分别用于评估纳入的随机对照试验(RCTs)和观察性研究的偏倚风险。

Due to excess heterogeneity, results were synthesised qualitatively.

由于异质性过高，结果进行了定性综合。

Results

This SR included 42 studies for efficacy and safety (n = 1798 patients), 13 for tapering, and 31 for amyloidosis.

本系统评价纳入了42项研究以评估疗效和安全性（共1798名患者），13项研究关于减量，以及31项研究关于淀粉样变性。

Based on 6 RCTs of interleukin (IL)-1 blockers and observational studies, these biologicals seem to be effective in decreasing the number of attacks and acute-phase reactants and improving disease activity scores and patient-reported outcomes.

基于6项关于白细胞介素(IL)-1阻断剂的随机对照试验(RCTs)和观察性研究，这些生物制剂似乎在减少发作次数和急性期反应物、改善疾病活动评分和患者报告的结果方面是有效的。

They also seem relatively safe.

它们似乎也相对安全。

An RCT showed the equivalence of once-daily vs twice-daily doses of colchicine.

一项随机对照试验显示，每日一次与每日两次剂量的秋水仙碱具有等效性。

The evidence of AA amyloidosis was entirely observational but reassuring for a deadly complication.

AA淀粉样变性的证据完全是观察性的，但对于这种致命并发症来说是令人安心的。

Conclusions

Biological agents, particularly IL-1 inhibitors, are effective and relatively safe options for FMF patients who do not respond to colchicine.

生物制剂，特别是IL-1抑制剂，对于那些对秋水仙碱无效的家族性地中海热（FMF）患者来说是有效且相对安全的选择。

These treatments may be promising alternatives for managing symptoms and preventing comorbidities in both paediatric and adult populations.

这些治疗可能对于管理症状和预防儿童和成人人群中的合并症是前景看好的替代方案。