S1P受体调节剂治疗复发型多发性硬化的有效性及安全性的真实世界研究  

Effectiveness and safety of S1P receptor modulators in patients with relapsing multiple sclerosis

作　　者：陈欣[1] 张烁琪 宋文慧 秦川[1] 田代实[1] 潘邓记[1] 肖君[1] Xin;Zhang Shuoqi;Song Wenhui(Department of Neurology,Tongji Hospital,Tongji Medical College,Huazhong University of Science and Technology,Wuhan 430030)

机构地区：[1]华中科技大学同济医学院附属同济医院神经内科、华中科技大学神经损伤与功能重建湖北省重点实验室,武汉430030 [2]华中科技大学同济医学院附属同济医院放射科,武汉430030

出　　处：《卒中与神经疾病》2023年第3期297-301,共5页Stroke and Nervous Diseases

摘　　要：目的评估鞘氨醇1-磷酸(Sphingosine 1-phosphate,S1P)受体调节剂在真实世界中对复发型多发性硬化(Relapsing multiple sclerosis,RMS)患者的有效性和安全性。方法这是一项多中心、回顾性真实世界研究。本研究回顾性地收集和汇总了湖北省2年内(2020年5月1日0-2022年4月30日)口服S1P受体调节剂(西尼莫德或芬戈莫德)的RMS患者的临床及影像学数据;主要终点为年化复发率(Annualized relapse rate,ARR),次要终点是末次随访时扩展残疾量表(Expanded disability status scale,EDSS)评分的变化、末次随访无复发患者的比例以及S1P受体调节剂的安全性和耐受性。结果本研究共纳入104例RMS患者(芬戈莫德31例,西尼莫德73例),其中男37例(35.6%),女67例(64.4%),平均年龄(34.4±1.2)岁,确诊多发性硬化(Multiple sclerosis,MS)平均时间为(4.3±0.4)年,中位随访时间为10(2~24)个月,接受S1P受体调节剂治疗的中位时间为7.5(1~24)个月,22例患者(21.2%)既往有疾病修饰治疗(Disease modifying therapy,DMT)用药史。S1P受体调节剂可以显著降低RMS患者的ARR[治疗后(0.03±0.17)%,治疗前(1.23±1.13)%,P<0.001],降幅达97%。末次随访时患者EDSS评分较基线明显下降[末次随访(1.11±1.67)分,治疗前(2.22±2.13)分,P<0.001]。97.1%的患者在研究期间维持无复发。随访过程中27例患者出现不良反应,最常见的不良反应包括肝酶升高、淋巴细胞计数减少,无严重不良事件的发生。结论本真实世界研究证实,S1P受体调节剂能显著减少RMS患者的复发,延缓残疾进展,且安全性较好。Objective To assess the effectiveness and safety of S1P receptor modulators in a cohort of RMS patients in the real world.Methods It was a multicenter,retrospective,observational,real-world study.This study retrospectively collected and summarized the clinical and imaging data of RMS patients who took oral S1P receptor modulators(Siponimod and Fingolimod)within 2 years(2020.5.1 to 2022.4.30)in Hubei Province.The primary endpoint was evaluated by the change in annualized recurrence rate(ARR).The secondary endpoints were the change in EDSS scores,the proportion of relapse-free patients at the last follow-up visit and the safety and tolerability of S1P receptor modulators.Results A total of 104 patients with RMS(31 patients with Fingolimod and 73 patients with Siponimod)were enrolled in this study.There were 37 males(35.6%)and 67 females(64.4%),with an average age of(34.4±1.2)years.The time since MS diagnosis was(4.3±0.4)years.The median follow-up time was 10(2~24)months and the median duration of S1P receptor modulator therapy was 7.5(1~24)months.22 persons(21.2%)had received previous disease modifying therapies(DMTs)at any time.The main ARR was significantly lower at the last follow-up visit compared to that before S1P receptor modulator treatment[post-treatment(0.03±0.17)%vs pre-treatment(1.23±1.13)%,P<0.001]with a drop of 97 percent.At the last follow-up visit,the EDSS scores decreased significantly from baseline[(1.11±1.67)points at last follow-up vs pre-treatment(2.22±2.13)points,P<0.001].Up to 97.1%of patients remained recurrence-free during the data collection period.Among the 27 adverse events(AEs),the most common AEs were elevated liver enzymes and decreased lymphocyte count,and no serious adverse events occurred.Conclusion This real-world study confirmed that S1P receptor modulators could significantly reduce recurrence and delay the progression of disability in patients with RMS and they were well tolerated and no new safety findings were observed in this study.

关 键 词：复发型多发性硬化 鞘氨醇1-磷酸受体调节剂 年化复发率 扩展残疾量表评分 

分 类 号：R744.51[医药卫生—神经病学与精神病学]