Pagnoux C, Carette S, Khalidi N A, Walsh M, Hiemstra T F, Cuthbertson D, Langford C, Hoffman G, Koening C L, Monach P A, Moreland L, Mouthon L, Seo P, Specks U, Ytterberg S, Westman K, Hoglund P, Harper L, Flossman O, Luqmani R, Savage C O, Rasmussen N, de Groot K, Tesar V, Jayne D, Merkel P A, Guillevin L
Division of Rheumatology, University of Toronto, Canada, and Department of Internal Medicine, National Referral Center for Necrotising Vasculitides and Systemic Sclerosis, Department of Internal Medicine, Hôpital Cochin, University of Paris, France.
Division of Rheumatology, University of Toronto, Canada.
Clin Exp Rheumatol. 2015 Mar-Apr;33(2 Suppl 89):S-77-83. Epub 2015 May 26.
To analyse the differences between patients with granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) entered into randomised clinical trials (RCTs) and those followed in large observational cohorts.
The main characteristics and outcomes of patients with generalised and/or severe GPA or MPA with a five-factor score ≥ 1 enrolled in the French Vasculitis Study Group (FVSG) or the US-Canadian-based Vasculitis Clinical Research Consortium cohorts were compared to those enrolled in one of 2 FVSG clinical RCTs (WEG91, WEGENT) or 3 European Vasculitis Society clinical trials (CYCLOPS, CYCAZAREM, IMPROVE).
657 patients (65.3% with GPA) in RCTs were compared to 437 in cohorts (90.6% with GPA). RCT patients were older at diagnosis than the cohort patients (56.6 ± 13.9 vs. 46.8 ± 17.3 years), had higher Birmingham vasculitis activity score (19.5 ± 9.1 vs. 16.9 ± 7.4), and more frequent kidney disease (84.0% vs. 54.9%) but fewer ear, nose, and throat symptoms (56.8% vs. 72.2%). At 56 months post-diagnosis, mortality and relapse rates, adjusted for age and renal function, were higher for patients with GPA in RCTs vs. cohorts (10.7% vs. 2.5% [p=0.001] and 22.5% vs. 15.6% [p=0.03], respectively) but similar for patients with MPA (6.2% vs. 6.6% [p=0.92] and 16.6% vs. 10.1% [p=0.39], respectively).
Patients with GPA or MPA in RCTs and those in observational cohorts show important differences that should be remembered when interpreting results based on these study populations.
分析纳入随机临床试验（RCT）的肉芽肿性多血管炎（GPA）或显微镜下多血管炎（MPA）患者与大型观察性队列中患者之间的差异。
将法国血管炎研究组（FVSG）或美国 - 加拿大血管炎临床研究联盟队列中纳入的五因素评分≥1的全身性和/或重度GPA或MPA患者的主要特征和结局，与纳入两项FVSG临床RCT（WEG91、WEGENT）之一或三项欧洲血管炎学会临床试验（CYCLOPS、CYCAZAREM、IMPROVE）的患者进行比较。
将RCT中的657例患者（65.3%为GPA）与队列中的437例患者（90.6%为GPA）进行比较。RCT患者诊断时年龄比队列患者大（56.6±13.9岁对46.8±17.3岁），伯明翰血管炎活动评分更高（19.5±9.1对16.9±7.4），肾脏疾病更常见（84.0%对54.9%），但耳、鼻、喉症状较少（56.8%对72.2%）。诊断后56个月，经年龄和肾功能调整后，RCT中GPA患者的死亡率和复发率高于队列患者（分别为10.7%对2.5%[p = 0.001]和22.5%对15.6%[p = 0.03]），但MPA患者相似（分别为6.2%对6.6%[p = 0.92]和16.6%对10.1%[p = 0.39]）。
RCT中的GPA或MPA患者与观察性队列中的患者存在重要差异，在基于这些研究人群解释结果时应予以牢记。
