Clinical Characteristics of an Internet-Based Cohort of Patient-Reported Diagnosis of Granulomatosis With Polyangiitis and Microscopic Polyangiitis: Observational Study
Clinical Characteristics of an Internet-Based Cohort of Patient-Reported Diagnosis of Granulomatosis With Polyangiitis and Microscopic Polyangiitis: Observational Study
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DOI：
10.2196/17231
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发表时间：
2020-07-20
影响因子：
7.4
通讯作者：
Merkel, Peter A.
中科院分区：
文献类型：
--
作者：
Springer, Jason Michael;Kermani, Tanaz A.;Merkel, Peter A.
背景：利用传统的卓越中心方法进行涉及罕见疾病的临床试验仍然具有挑战性。以患者为基础的登记已被证明是可行的和有效的其他diseases.Objective：本报告概述了一个大型的互联网登记队列的参与者与自我报告的诊断肉芽肿性多血管炎或显微镜下多血管炎的临床特征。基于互联网的前瞻性纵向队列中自我报告诊断为肉芽肿伴多血管炎或显微镜下多血管炎的患者（来自血管炎患者动力研究网络）。症状，诊断测试和治疗的数据收集使用标准化questionnaire.Results：该研究比较了肉芽肿性多血管炎（n=762）和显微镜下多血管炎（n=164）的患者。在该队列中，97.7%（904/925）报告诊断已由医生确认。与显微镜下多血管炎相比，肉芽肿性多血管炎患者报告的耳、鼻和咽喉表现明显更多（肉芽肿性多血管炎：641/723，88.7%;显微镜下多血管炎：89/164，54.3%; z=10.42，P
Background: Utilizing the traditional centers of excellence approach to conduct clinical trials involving rare diseases remains challenging. Patient-based registries have been shown to be both feasible and valid in several other diseases.Objective: This report outlines the clinical characteristics of a large internet registry cohort of participants with a self-reported diagnosis of granulomatosis with polyangiitis or microscopic polyangiitis.Methods: Patients with a self-reported diagnosis of granulomatosis with polyangiitis or microscopic polyangiitis in an internet-based prospective longitudinal cohort (from the Vasculitis Patient-Powered Research Network) were included. Data on symptoms, diagnostic testing, and treatment were collected using standardized questionnaires.Results: The study compared patients with granulomatosis with polyangiitis (n=762) and patients with microscopic polyangiitis (n=164). Of the cohort, 97.7% (904/925) reported the diagnosis had been confirmed by a physician. Compared to microscopic polyangiitis, patients with granulomatosis with polyangiitis reported significantly more ear, nose, and throat manifestations (granulomatosis with polyangiitis: 641/723, 88.7%; microscopic polyangiitis: 89/164, 54.3%; z=10.42, P