【摘要】 目的 系统评价阿伐可泮治疗抗中性粒细胞胞质抗体相关性血管炎（anti-neutrophil cytoplasmic antibody-associated vasculitis,AAV）的疗效和安全性。方法 通过计算机检索Embase、PubMed、Cochrane Library、Web of Science、中国知网、万方及维普中英文数据库上关于阿伐可泮治疗AAV的随机对照试验（randomized controlled trial,RCT）及队列研究，检索时限均从建库至2025年6月。采用RevMan 5.4软件进行Meta分析。结果 最终共纳入10篇文献，包括3项RCT、7项队列研究，总样本量727例。Meta分析结果显示，疗效方面，RCT研究中阿伐可泮治疗AAV的缓解率与对照组相比，差异无统计学意义（RR=1.03,95%CI:0.90～1.18,P=0.690），持续缓解率优于对照组，且差异具有统计学意义（RR=1.22,95%CI:1.02～1.45,P=0.030），对照研究显示阿伐可泮组的复发率显著低于对照组，差异具有统计学意义（RR=0.49,95%CI:0.30～0.79,P=0.004）；单臂队列研究显示阿伐可泮治疗AAV的缓解率为88%（95%CI:0.82～0.92），持续缓解率为87%（95%CI:0.78～0.93），复发率为7%（95%CI:0.04～0.12）。肾脏评价方面，RCT中估算肾小球滤过率（estimated glomerular filtration rate,eGFR）[mL·min^-1·（1.73 m²）^-1]从基线至终点的变化与对照组相比，差异具有统计学意义（MD=2.76,95%CI:0.21～5.30,P=0.03）；在真实世界研究中，阿伐可泮治疗AAV患者6个月（MD=9.40,95%CI:0.44～18.37,P=0.04）和12个月（MD=13.88,95%CI:8.47～19.28,P<0.01）时eGFR与基线相比差异也具有统计学意义（P<0.05）。不良反应方面，总不良事件发生率（RR=1.00,95%CI:0.96～1.03,P=0.79）及感染率（RR=0.93,95%CI:0.81～1.07,P=0.30）与对照组比较，差异无统计学意义（P>0.05）；在单臂队列研究中阿伐可泮治疗AAV的消化道不良反应发生率为5%（95%CI:0.03～0.09），但转氨酶升高的发生率在日本人群39%（95%CI:0.26～0.55）中高于非日本人群4%（95%CI:0.02～0.08）。结论 阿伐可泮作为AAV的辅助治疗，可提高持续缓解率、降低复发率并改善肾功能，且总体安全性良好。但需注意肝功能监测，尤其是在日本人群中，未来需更多长期随访研究以进一步验证其疗效和安全性。更多还原

【Abstract】 Objective To systematically evaluate the efficacy and safety of avacopan in patients with anti-neutrophil cytoplasmic antibody-associated vasculitis（AAV）.Methods We performed a comprehensive literature search of Embase,PubMed,the Cochrane Library,Web of Science,CNKI,Wanfang,and VIP from database inception to June 2025 for randomized controlled trials（RCTs） and cohort studies assessing avacopan in AAV.Data were pooled using RevMan 5.4.Results Ten studies（3RCTs and 7 cohort studies） comprising 727 patients were included.In RCTs,avacopan did not significantly affect overall remission rate compared with control（RR=1.03;95%CI:0.90-1.18;P=0.69）,but it significantly improved sustained remission rate（RR=1.22;95%CI:1.02-1.45;P=0.03）.Cohort studies showed a lower relapse rate with avacopan versus control（RR=0.49;95%CI:0.30-0.79;P=0.004）.Single-arm pooled analyses indicated high event rates for remission（88%;95%CI:0.82-0.92） and sustained remission（87%;95%CI:0.78-0.93）,with a low pooled relapse proportion（7%;95%CI:0.04-0.12）.Renal outcomes favored avacopan:RCTs showed a small but statistically significant improvement in eGFR [mL·min^-1·（1.73 m²）^-1] change from baseline to endpoint（MD=2.76;95%CI:0.21-5.30;P=0.03）.Real-world cohort data demonstrated clinically meaningful eGFR gains at 6 months（MD=9.40;95%CI:0.44-18.37;P=0.04） and 12 months（MD=13.88;95%CI:8.47-19.28;P<0.01）versus baseline.Safety:Overall adverse event rates（RR=1.00;95%CI:0.96-1.03;P=0.79） and infection rates（RR=0.93;95%CI:0.81-1.07;P=0.30） were comparable to controls.Pooled incidence from single-arm cohorts showed a low rate of gastrointestinal adverse events（5%;95%CI:0.03-0.09）.Elevated transaminases were notably more common in Japanese patients（39%;95%CI:0.26-0.55） than in non-Japanese populations（4%;95%CI:0.02-0.08）.Conclusion Avacopan,used as adjunctive therapy for AAV,is associated with improved sustained remission,reduced relapse,and improved renal function,and its overall safety profile is acceptable.However,liver enzyme monitoring is advisable,particularly in Japanese patients.Additional large-scale and long-term studies are warranted to confirm these findings.更多还原