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瑞士多发性硬化症队列研究(SMSC):一项对瑞士范围内疾病演变关键阶段和新治疗选择的前瞻性调查。

The Swiss Multiple Sclerosis Cohort-Study (SMSC): A Prospective Swiss Wide Investigation of Key Phases in Disease Evolution and New Treatment Options.

作者信息

Disanto Giulio, Benkert Pascal, Lorscheider Johannes, Mueller Stefanie, Vehoff Jochen, Zecca Chiara, Ramseier Simon, Achtnichts Lutz, Findling Oliver, Nedeltchev Krassen, Radue Ernst-Wilhelm, Sprenger Till, Stippich Christoph, Derfuss Tobias, Louvion Jean-François, Kamm Christian P, Mattle Heinrich P, Lotter Christoph, Du Pasquier Renaud, Schluep Myriam, Pot Caroline, Lalive Patrice H, Yaldizli Özgür, Gobbi Claudio, Kappos Ludwig, Kuhle Jens

机构信息

Department of Neurology, Regional Hospital Lugano (EOC), Lugano, Switzerland.

Clinical Trial Unit, University Hospital Basel, Switzerland.

出版信息

PLoS One. 2016 Mar 31;11(3):e0152347. doi: 10.1371/journal.pone.0152347. eCollection 2016.

Abstract

The mechanisms leading to disability and the long-term efficacy and safety of disease modifying drugs (DMDs) in multiple sclerosis (MS) are unclear. We aimed at building a prospective cohort of MS patients with standardized collection of demographic, clinical, MRI data and body fluids that can be used to develop prognostic indicators and biomarkers of disease evolution and therapeutic response. The Swiss MS Cohort (SMSC) is a prospective observational study performed across seven Swiss MS centers including patients with MS, clinically isolated syndrome (CIS), radiologically isolated syndrome or neuromyelitis optica. Neurological and radiological assessments and biological samples are collected every 6-12 months. We recruited 872 patients (clinically isolated syndrome [CIS] 5.5%, relapsing-remitting MS [RRMS] 85.8%, primary progressive MS [PPMS] 3.5%, secondary progressive MS [SPMS] 5.2%) between June 2012 and July 2015. We performed 2,286 visits (median follow-up 398 days) and collected 2,274 serum, plasma and blood samples, 152 cerebrospinal fluid samples and 1,276 brain MRI scans. 158 relapses occurred and expanded disability status scale (EDSS) scores increased in PPMS, SPMS and RRMS patients experiencing relapses. Most RRMS patients were treated with fingolimod (33.4%), natalizumab (24.5%) or injectable DMDs (13.6%). The SMSC will provide relevant information regarding DMDs efficacy and safety and will serve as a comprehensive infrastructure available for nested research projects.

摘要

导致多发性硬化症(MS)患者残疾的机制以及疾病修饰药物(DMDs)的长期疗效和安全性尚不清楚。我们旨在建立一个MS患者的前瞻性队列,标准化收集人口统计学、临床、MRI数据和体液,以用于开发疾病进展和治疗反应的预后指标和生物标志物。瑞士MS队列(SMSC)是一项在七个瑞士MS中心进行的前瞻性观察性研究,纳入了MS、临床孤立综合征(CIS)、放射学孤立综合征或视神经脊髓炎患者。每6 - 12个月进行一次神经学和放射学评估并收集生物样本。我们在2012年6月至2015年7月期间招募了872名患者(临床孤立综合征[CIS]占5.5%,复发缓解型MS[RRMS]占85.8%,原发进展型MS[PPMS]占3.5%,继发进展型MS[SPMS]占5.2%)。我们进行了2286次随访(中位随访时间398天),收集了2274份血清、血浆和血液样本、152份脑脊液样本以及1276次脑部MRI扫描。158次复发发生,复发的PPMS、SPMS和RRMS患者的扩展残疾状态量表(EDSS)评分增加。大多数RRMS患者接受了芬戈莫德(33.4%)、那他珠单抗(24.5%)或注射用DMDs(13.6%)治疗。SMSC将提供有关DMDs疗效和安全性的相关信息,并将作为可供嵌套研究项目使用的综合基础设施。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4fbd/4816556/a8140bb2cbfa/pone.0152347.g001.jpg

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