University of Kansas Medical Center, Kansas City, KS, United States.
Division of Rheumatology, Department of Medicine, University of California, Santa Monica, CA, United States.
J Med Internet Res. 2020 Jul 20;22(7):e17231. doi: 10.2196/17231.
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.
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.
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.
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<.001), fevers (granulomatosis with polyangiitis: 325/588, 55.3%; microscopic polyangiitis: 64/139, 46.0%; z=1.96, P=.05), joint involvement (granulomatosis with polyangiitis: 549/688, 79.8%; microscopic polyangiitis: 106/154, 68.8%; z=2.96, P=.003), and pulmonary involvement (granulomatosis with polyangiitis: 523/734, 71.3%; microscopic polyangiitis: 90/154, 58.4%; z=3.13, P=.002). Compared to microscopic polyangiitis, patients with granulomatosis with polyangiitis reported significantly less renal involvement (granulomatosis with polyangiitis: 457/743, 61.5%; microscopic polyangiitis: 135/163, 82.8%; z=-5.18, P<.001) and renal transplantation (granulomatosis with polyangiitis: 10/721, 1.4%; microscopic polyangiitis: 7/164, 4.3%; z=-2.43, P=.02). Antineutrophil cytoplasmic antibody positivity was reported in 94.2% (652/692) of patients with granulomatosis with polyangiitis and 96.1% (147/153) of patients with microscopic polyangiitis. A biopsy showing vasculitis was reported in 77.0% (562/730) of patients with granulomatosis with polyangiitis and 81.9% (131/160) of patients with microscopic polyangiitis.
In this large, internet-based cohort of patients with a self-reported diagnosis of granulomatosis with polyangiitis or microscopic polyangiitis, disease manifestations were consistent with expectations for each type of vasculitis. Given the rarity of these and other vasculitides, conducting some types of research through internet-based registries may provide an efficient alternative to inperson, center-of-excellence clinical trials.
利用传统的卓越中心方法来开展涉及罕见疾病的临床试验仍然具有挑战性。在其他几种疾病中，已经证明基于患者的登记处既可行又有效。
本报告概述了一项大型互联网注册队列中自我报告患有肉芽肿性多血管炎或显微镜下多血管炎的参与者的临床特征。
纳入了在互联网前瞻性纵向队列（来自血管炎患者驱动的研究网络）中自我报告患有肉芽肿性多血管炎或显微镜下多血管炎的患者。使用标准化问卷收集症状、诊断检测和治疗数据。
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）患者。该队列中，97.7%（904/925）的患者报告其诊断已由医生确认。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部、鼻部和喉部表现明显更多（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<.001）、发热（肉芽肿性多血管炎：325/588，55.3%；显微镜下多血管炎：64/139，46.0%；z=1.96，P=.05）、关节受累（肉芽肿性多血管炎：549/688，79.8%；显微镜下多血管炎：106/154，68.8%；z=2.96，P=.003）和肺部受累（肉芽肿性多血管炎：523/734，71.3%；显微镜下多血管炎：90/154，58.4%；z=3.13，P<.001）。与显微镜下多血管炎相比，肉芽肿性多血管炎患者报告的肾脏受累明显较少（肉芽肿性多血管炎：457/743，61.5%；显微镜下多血管炎：135/163，82.8%；z=-5.18，P<.001）和肾移植（肉芽肿性多血管炎：10/721，1.4%；显微镜下多血管炎：7/164，4.3%；z=-2.43，P=.02）。94.2%（652/692）的肉芽肿性多血管炎患者和 96.1%（147/153）的显微镜下多血管炎患者报告抗中性粒细胞胞质抗体阳性。77.0%（562/730）的肉芽肿性多血管炎患者和 81.9%（131/160）的显微镜下多血管炎患者报告了血管炎活检。
在这项基于互联网的自我报告患有肉芽肿性多血管炎或显微镜下多血管炎的大型队列中，疾病表现与每种血管炎的预期相符。鉴于这些和其他血管炎的罕见性，通过基于互联网的登记处开展某些类型的研究可能是一种替代卓越中心临床试验的有效方法。
