在抗中性粒细胞胞质抗体相关性血管炎日本患者中的有效性和安全性：一项随机 3 期研究的亚分析。

Efficacy and safety of avacopan in Japanese patients with antineutrophil cytoplasmic antibody-associated vasculitis: A subanalysis of a randomized Phase 3 study.

机构信息

Division of Rheumatology, Department of Internal Medicine, Tokyo Women's Medical University School of Medicine, Tokyo, Japan.

Department of Nephrology and Rheumatology, Kyorin University School of Medicine, Tokyo, Japan.

出版信息

Mod Rheumatol. 2023 Mar 2;33(2):338-345. doi: 10.1093/mr/roac037.

DOI:10.1093/mr/roac037

PMID:35482532

Abstract

OBJECTIVES

This subgroup analysis of the randomized, double-blind, Phase 3 ADVOCATE study evaluated the efficacy and safety of avacopan compared with tapered prednisone in Japanese patients with antineutrophil cytoplasmic antibody-associated vasculitis.

METHODS

Patients with microscopic polyangiitis (MPA) or granulomatosis with polyangiitis (GPA) received either avacopan 30 mg twice daily for 52 weeks plus prednisone-matching placebo or tapered prednisone over 20 weeks plus avacopan-matching placebo for 52 weeks. The two primary efficacy endpoints were clinical remission at Week 26 and sustained remission at Week 52.

RESULTS

Compared with the overall population (N = 330), Japanese patients (N = 21) were older and had worse renal function, and a higher proportion were female and had MPA. The proportion of Japanese patients with clinical remission at Week 26 was 9/11 (81.8%) with avacopan vs. 7/10 (70.0%) with prednisone (overall population: 72.3% vs. 70.1%) and with sustained remission at Week 52 was 8/11 (72.7%) vs. 4/10 (40.0%), respectively (overall population: 65.7% vs. 54.9%). The safety profile of avacopan was similar in Japanese patients and the overall study population.

CONCLUSIONS

The efficacy and safety of avacopan in Japanese patients with MPA or GPA were comparable to that observed in the overall ADVOCATE study population.

摘要

目的

本项 ADVOCATE 研究的随机、双盲、3 期亚组分析评估了与逐渐减量的泼尼松龙相比，阿伐考帕在日本抗中性粒细胞胞质抗体相关性血管炎患者中的疗效和安全性。

方法

患有显微镜下多血管炎（MPA）或肉芽肿性多血管炎（GPA）的患者接受阿伐考帕 30mg，每日两次，共 52 周，同时给予泼尼松龙匹配安慰剂；或接受逐渐减量的泼尼松龙，共 20 周，同时给予阿伐考帕匹配安慰剂，共 52 周。主要疗效终点为第 26 周的临床缓解和第 52 周的持续缓解。

结果

与总体人群（N=330）相比，日本患者（N=21）年龄更大，肾功能更差，女性比例更高，且 MPA 患者比例更高。第 26 周时，阿伐考帕组的临床缓解率为 9/11（81.8%），泼尼松龙组为 7/10（70.0%）（总体人群：72.3% vs. 70.1%）；第 52 周时，阿伐考帕组的持续缓解率为 8/11（72.7%），泼尼松龙组为 4/10（40.0%）（总体人群：65.7% vs. 54.9%）。日本患者和总体研究人群的阿伐考帕安全性特征相似。