Doubelt Irena, Springer Jason M, Kermani Tanaz A, Sreih Antoine G, Burroughs Cristina, Cuthbertson David, Carette Simon, Khalidi Nader A, Koening Curry L, Langford Carol, McAlear Carol A, Moreland Larry W, Monach Paul A, Shaw Dianne G, Seo Philip, Specks Ulrich, Warrington Kenneth J, Young Kalen, Merkel Peter A, Pagnoux Christian
Vasculitis Clinic, Mount Sinai Hospital, Toronto, ON, Canada.
Division of Rheumatology, University of Toronto, Toronto, ON, Canada.
Interact J Med Res. 2022 May 25;11(1):e27273. doi: 10.2196/27273.
Patient-based registries can help advance research on rare diseases such as eosinophilic granulomatosis with polyangiitis (EGPA), a complex multiorgan form of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis.
The aim of this study is to compare patient-reported and physician-reported data on manifestations, treatments, and outcomes for patients with EGPA.
We completed a comparative analysis of patients ≥18 years with EGPA in Canada and the United States from the following 2 cohorts: (1) The Vasculitis Patient-Powered Research Network (VPPRN), a self-enrolled secure portal with patient-entered data updated quarterly (2014-2019) and (2) the Vasculitis Clinical Research Consortium (VCRC) observational studies, a physician-entered database (2003-2019) of patients who fulfilled the 1990 American College of Rheumatology classification criteria for EGPA. The studied parameters included demographic characteristics, clinical manifestations, ANCA status, treatments, and relapses.
Data from 195 patients with a validated diagnosis of EGPA in the VPPRN and 354 patients enrolled in the VCRC were analyzed. Compared to the VCRC cohort, the patients in the VPPRN cohort were more likely to be female (135/195, 69.2% compared to 209/354, 59%; P=.02) and younger at diagnosis (47.3 compared to 50.0 years; P=.03); both cohorts reported similar frequencies of asthma (177/184, 96.2% in the VPPRN cohort compared to 329/354, 92.9% in the VCRC cohort; P=.13) and cardiac manifestations (44/153, 28.8% compared to 75/354, 21.2%; P=.06), but the VPPRN cohort reported less frequent lung manifestations other than asthma and more frequent disease manifestations in all other organ systems. The ANCA positivity was 48.9% (64/131) in the VPPRN patients compared to 38.9% (123/316; P=.05) in the VCRC cohort. Relapsing disease after study enrollment was reported in 32.3% (63/195) of patients in the VPPRN compared to 35.7% (99/277) of patients in the VCRC. Most therapies (GC, cyclophosphamide, mepolizumab) were used at similar frequencies in both groups, except for rituximab with VPPRN patients reporting more use than the VCRC cohort (47/195, 24.1% compared to 29/277, 10.5%; P<.001).
Overall, patients and physicians report manifestations of EGPA at similar frequencies. However, observed differences between patient and physician reports imply the potential occurrence of selection biases. These results support the use of patient-reported data in EGPA but also the need for careful consideration of disease-specific definitions for the study of EGPA and how patient- and physician-reported data are collected.
ClinicalTrials.gov NCT00315380, https://clinicaltrials.gov/ct2/show/NCT00315380; ClinicalTrials.gov NCT01241305, https://clinicaltrials.gov/ct2/show/NCT01241305.
基于患者的登记系统有助于推进对罕见疾病的研究，如嗜酸性肉芽肿性多血管炎（EGPA），这是一种抗中性粒细胞胞浆抗体（ANCA）相关血管炎的复杂多器官形式。
本研究旨在比较EGPA患者的患者报告数据与医生报告数据，涉及临床表现、治疗及结局。
我们对加拿大和美国年龄≥18岁的EGPA患者进行了比较分析，数据来自以下两个队列：（1）血管炎患者驱动研究网络（VPPRN），这是一个患者自行登记的安全门户，患者输入的数据每季度更新一次（2014 - 2019年）；（2）血管炎临床研究联盟（VCRC）的观察性研究，这是一个医生输入的数据库（2003 - 2019年），纳入了符合1990年美国风湿病学会EGPA分类标准的患者。研究参数包括人口统计学特征、临床表现、ANCA状态、治疗及复发情况。
对VPPRN中195例经确诊的EGPA患者和VCRC中354例患者的数据进行了分析。与VCRC队列相比，VPPRN队列中的患者更可能为女性（135/195，69.2% 对比209/354，59%；P = 0.02）且诊断时年龄更小（47.3岁对比50.0岁；P = 0.03）；两个队列报告的哮喘发生率相似（VPPRN队列中177/184，96.2% 对比VCRC队列中329/354，92.9%；P = 0.13）以及心脏表现发生率相似（44/153，28.8% 对比75/354，21.2%；P = 0.06），但VPPRN队列报告除哮喘外的肺部表现频率较低，而在所有其他器官系统中的疾病表现频率较高。VPPRN患者的ANCA阳性率为48.9%（64/131），对比VCRC队列中的38.9%（123/316；P = 0.05）。VPPRN中32.3%（63/195）的患者报告在研究入组后疾病复发，对比VCRC中35.7%（99/277）的患者。除利妥昔单抗外，大多数治疗方法（糖皮质激素、环磷酰胺、美泊利单抗）在两组中的使用频率相似，VPPRN患者使用利妥昔单抗的比例高于VCRC队列（47/195，24.1% 对比29/277，10.5%；P < 0.001）。
总体而言，患者和医生报告的EGPA表现频率相似。然而，患者报告与医生报告之间观察到的差异意味着可能存在选择偏倚。这些结果支持在EGPA研究中使用患者报告的数据，但也需要仔细考虑EGPA研究中特定疾病的定义以及如何收集患者和医生报告的数据。
ClinicalTrials.gov NCT00315380，https://clinicaltrials.gov/ct2/show/NCT00315380；ClinicalTrials.gov NCT01241305，https://clinicaltrials.gov/ct2/show/NCT01241305。
