糖皮质激素与糖皮质激素加环磷酰胺治疗伴有不良预后因素的嗜酸性粒细胞肉芽肿伴多血管炎。

IF 7.9 1区医学 Q1 IMMUNOLOGY

Journal of autoimmunity Pub Date : 2024-11-16 DOI:10.1016/j.jaut.2024.103338

Boris Sorin , Matthias Papo , Renato A. Sinico , Vítor Silvestre Teixeira , Nils Venhoff , Maria-Letizia Urban , Michele Iudici , Juliane Mahrhold , Francesco Locatelli , Giulia Cassone , Franco Schiavon , Benjamin Seeliger , Thomas Neumann , Claudia Feder , Claus Kroegel , Matthieu Groh , Chiara Marvisi , Maxime Samson , Thomas Barba , David Jayne , Benjamin Terrier

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引用次数: 0

摘要

治疗目的现行指南建议用糖皮质激素（GCs）加环磷酰胺（CYC）联合治疗预后不良的嗜酸性粒细胞肉芽肿伴多血管炎（EGPA）。然而，几乎没有数据支持加用 CYC 的必要性。本研究的目的是比较糖皮质激素加 CYC 与单用糖皮质激素作为诱导疗法治疗预后不良的 EGPA：方法：我们利用欧洲多中心回顾性数据库中的观察数据模拟了一项目标试验。我们纳入了在1985年6月至2018年11月期间接受GCs或GCs加CYC治疗、1996年五因素评分（FFS）至少为1的新诊断EGPA患者。倾向评分分析用于调整潜在的混杂因素。主要结果是12个月时的复发。次要结果包括12个月时的严重复发和24个月时的GC依赖性哮喘和/或耳鼻喉（ENT）表现：结果：共纳入 209 名患者：47%的患者为男性，诊断时的平均年龄为52岁（±16岁）；26%的患者仅接受了GCs治疗，74%的患者接受了GCs加CYC治疗。经调整后，与单用 GCs 组相比，GCs 加 CYC 组的复发风险（危险比 [HR]：0.24，95%CI [0.08-0.67]，p = 0.007）、严重复发风险（HR：0.24，95%CI [0.07-0.85]，p = 0.026）以及 GC 依赖性哮喘和/或耳鼻喉表现的比例（几率比：0.30，95%CI [0.14-0.66]，p = 0.003）均较低：这项目标试验模拟研究表明，在使用 GCs 的基础上加用 CYC 可降低预后不良的 EGPA 患者血管炎复发的风险以及 GC 依赖性哮喘和/或耳鼻喉科表现的发生率。

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Glucocorticoids versus glucocorticoids plus cyclophosphamide in eosinophilic granulomatosis with polyangiitis with poor-prognosis factors

Objectives

Current guidelines suggest treating poor-prognosis eosinophilic granulomatosis with polyangiitis (EGPA) with a combination of glucocorticoids (GCs) plus cyclophosphamide (CYC). However, there is little data to support the need for the addition of CYC. The objective of this study was to compare GCs plus CYC to GCs alone as induction therapy in poor-prognosis EGPA.

Methods

We emulated a target trial using observational data from a European multicenter retrospective database. We included patients with newly diagnosed EGPA with a 1996 Five Factor Score (FFS) of at least 1, treated with GCs or GCs plus CYC between June 1985 and November 2018. Propensity score analysis was used to adjust for potential confounders. Primary outcome was relapse at 12months. Secondary outcomes included major relapse at 12months and GC-dependent asthma and/or ear nose and throat (ENT) manifestations at 24months.

Results

A total of 209 patients were included: 47 % were male and the mean age at diagnosis was 52 (±16 years); 26 % were treated with GCs alone and 74 % with GCs plus CYC. After adjustment, the risk of relapse (hazard ratio [HR]: 0.24, 95%CI [0.08–0.67], p = 0.007), major relapse (HR: 0.24, 95%CI [0.07–0.85], p = 0.026) and the proportion of GC-dependent asthma and/or ENT manifestations (odds ratio:0.30, 95%CI [0.14–0.66], p = 0.003) were lower in the GCs plus CYC group compared to the GCs alone group.

Conclusion

This target trial emulation study shows that the addition of CYC to GCs reduces the risk of vasculitis relapse and the rate of GC-dependent asthma and/or ENT manifestations in patients with poor-prognosis EGPA.

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来源期刊

Journal of autoimmunity 医学-免疫学

CiteScore

27.90

自引率

1.60%

发文量

117

审稿时长

17 days

期刊介绍： The Journal of Autoimmunity serves as the primary publication for research on various facets of autoimmunity. These include topics such as the mechanism of self-recognition, regulation of autoimmune responses, experimental autoimmune diseases, diagnostic tests for autoantibodies, as well as the epidemiology, pathophysiology, and treatment of autoimmune diseases. While the journal covers a wide range of subjects, it emphasizes papers exploring the genetic, molecular biology, and cellular aspects of the field. The Journal of Translational Autoimmunity, on the other hand, is a subsidiary journal of the Journal of Autoimmunity. It focuses specifically on translating scientific discoveries in autoimmunity into clinical applications and practical solutions. By highlighting research that bridges the gap between basic science and clinical practice, the Journal of Translational Autoimmunity aims to advance the understanding and treatment of autoimmune diseases.