Exploring outcomes and characteristics of myasthenia gravis: Rationale, aims and design of registry - The EXPLORE-MG registry
Exploring outcomes and characteristics of myasthenia gravis: Rationale, aims and design of registry - The EXPLORE-MG registry
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DOI：
10.1016/j.jns.2020.116830
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发表时间：
2020-07-15
影响因子：
4.4
通讯作者：
Nowak, Richard J.
中科院分区：
文献类型：
--
作者：
Anil, Rahul;Kumar, Aditya;Nowak, Richard J.
目的：虽然关于重症肌无力（MG）的诊断、治疗和流行病学有很多信息，但一个全面的数据登记和生物库对于更好地了解疾病机制、治疗结果和治疗策略的影响至关重要。我们的目的是设计和实施“重症肌无力的结果和特征探索（EXPLORE-MG）登记”来解决这些知识空白。方法：建立了一个基于网络的、非干预性的、纵向的、观察性的疾病和结局登记处；纳入NIH推荐的MG研究常用数据元素。根据预先规定的标准诊断为MG的个体有资格参加研究。一个补充性的生物资料库进一步加强了登记处。对2017年通过数据锁定收集的注册表数据进行了中期分析。结果：2011年至2017年在耶鲁大学MG诊所随访的MG患者共232例，其中总就诊记录2142次。232例MG患者（平均年龄60岁，17-99岁，男女比例为1.04:1），乙酰胆碱受体抗体阳性165例，肌肉特异性激酶抗体阳性20例，血清阴性47例。该队列包括64例眼部疾病患者，168例全身性疾病患者和65例胸腺切除术后患者，包括20例胸腺瘤相关MG患者。结论：识别可能预测治疗反应性或提供对患者结果的洞察的关键临床特征对于改善患者护理至关重要。由于目前的研究侧重于开发针对患者的靶向治疗方案，该登记处可以帮助提供来自长期随访的大型当代患者队列的重要临床和流行病学数据。
Objectives: Though much information exists about the diagnosis, treatment, and epidemiology of myasthenia gravis (MG), a comprehensive data registry and biorepository is critical to better understand disease mechanisms, treatment outcomes, and the impact of treatment strategies. We aimed to design and implement the "Exploring Outcomes and Characteristics of Myasthenia Gravis (EXPLORE-MG) Registry" to address these knowledge gaps.Methods: A web-based, non-interventional, longitudinal, observational disease and outcomes registry was developed; incorporating NIH recommended common data elements for the study of MG. Individuals diagnosed with MG based on prespecified criteria were eligible to participate. The registry was further strengthened by a complementary biorepository. An interim analysis was completed on registry data collected through data-lock in 2017.Results: A total of 232 MG patients, followed at the Yale MG Clinic from 2011 to 2017, were enrolled, which included 2142 total visit entries. Of the 232 MG patients (mean age 60 years, range 17-99; female:male, 1.04:1), 165 were acetylcholine receptor antibody-positive, 20 were muscle-specific kinase antibody-positive, and 47 were seronegative. This cohort consisted of 64 patients with ocular disease, 168 patients with generalized disease, and 65 patients post-thymectomy, including 20 with thymoma-associated MG.Conclusions: Identification of key clinical features that may predict treatment responsiveness or provide insight into patient outcomes is essential to improve patient care. As current research focuses on the development of patient-tailored, targeted-treatment regimens, this registry can help provide important clinical and epidemiological data from a large contemporary patient cohort with long-term follow-up.