目的:特发性炎性肌病(IIM)很少见,其特征是异质性表现和临床轨迹。利用远程研究方法有可能改善参与者的招募并增进对疾病的了解。我们旨在评估整个美国IIM患者的疾病特征,并比较通过移动应用程序或网站远程招募的患者与在肌炎诊所本地招募的患者之间的这些参数。
方法:“肌炎患者中心远程研究”(MyPACER)是一项针对美国IIM受试者的多中心前瞻性观察研究,通过传统的面对面诊所访问(基于中心的队列[CBC])竞争性招募,并远程使用移动应用程序或网站和社交媒体(远程研究队列[TRC])。数据收集包括基线人口统计学和临床变量,包括症状,器官受累,诊断测试结果和药物使用。
结果:该研究包括120名IIM患者,82在TRC和38在CBC。平均年龄为55±13.4,女性占75%,白种人占81%。两个队列都表现出相似的人口统计学特征。总的来说,41%皮肌炎,27%多发性肌炎,23%抗合成酶综合征,9%的坏死性肌炎患者入组,队列中亚型患病率相当(p=0.85)。这些群体在多种临床因素上表现出相似性,包括肌肉酶,诊断延迟,就业状况,各种患者和医生报告的结果,功能测试,以及胸部CT异常发现的频率,肺功能检查,和肌电图。TRC患者接受生物制剂和csDMARD的频率更高(分别为p<0.001和p=0.013)。
结论:远程研究招募产生的患者队列类似于传统的人口统计学和临床招募患者,表明其在临床研究中对稳健和多样化的患者招募的有效性。
OBJECTIVE: Idiopathic Inflammatory Myopathies (IIM) are rare and characterized by heterogeneous manifestations and clinical trajectories. Utilizing tele-research methods has the potential to improve participant recruitment and advance the understanding of the disease. We aimed to evaluate disease characteristics in IIM patients throughout the U.S. and compare these parameters between patients recruited remotely through mobile application or website vs those recruited locally in myositis clinics.
METHODS: \"Myositis Patient Centered Tele-Research\" (My PACER) is a multicentre prospective observational study of U.S. IIM subjects, competitively recruited through traditional in-person clinic visits (Center-Based Cohort [CBC]), and remotely using mobile application or website and social media (Tele-Research Cohort [TRC]). Data collection comprised baseline demographic and clinical variables, encompassing symptoms, organ involvement, diagnostic tests results and medication use.
RESULTS: The study included 120 IIM patients, 82 in the TRC and 38 in the CBC. The average age was 55 ± 13.4, 75% females and 81% Caucasians. Both cohorts exhibited similar demographic characteristics. Overall, 41% dermatomyositis, 27% polymyositis, 23% anti-synthetase syndrome, and 9% necrotizing myositis patients were enrolled, with comparable subtypes prevalence among cohorts (p= 0.85). The groups demonstrated similarities in multiple clinical factors, including muscle enzymes, diagnostic delay, employment status, various patient and physician-reported outcomes, functional tests, and the frequency of abnormal findings in chest CT, pulmonary function tests, and electromyography. TRC patients received biologics and csDMARDs more frequently (p< 0.001 and p= 0.013, respectively).
CONCLUSIONS: Tele-research recruitment yielded a patient cohort resembling traditionally recruited patients demographically and clinically, indicating its effectiveness for robust and diverse patient recruitment in clinical studies.