从真实世界看中国多发性硬化患者临床结局与医生治疗策略

Clinical outcomes and treatment strategy of multiple sclerosis in China:Results from a real-world cross-sectional Survey

目的评估中国多发性硬化(multiple sclerosis,MS)患者诊疗现状、临床结局及医生治疗策略。方法基于2021年6月至8月阿德尔菲(Adelphi)MS疾病特定计划^(TM)(DSP)关于中国不同区域医生(n=160)及其MS患者(n=956)横断面调查的资料,以诊疗现状、临床结局和医生治疗策略调整为研究终点进行二次数据分析。结果在缓解期治疗方面,仅30.1%(287例)患者接受疾病修正治疗(disease modifying treatment,DMT),且复发缓解型MS患者28.0%(215例)规范DMT占比显著低于原发进展型MS患者41.5%(34例)(P<0.05)。所有使用DMT的患者中,从确诊到开始DMT时间中位数(四分位数)为2.5(1.0,5.4)年,继发进展型患者开始最晚,为9.0(4.3,11.8)年。540例MS患者数据完整,其中过去12个月无疾病活动(no evidence of disease activity,NEDA)-3比例为27.4%。在治疗策略转化方面,患者复发1次、无复发但有残疾进展、无复发但磁共振成像表现有新病灶时,分别有42.2%、39.1%、62.1%患者的医生不调整用药。结论我国MS患者DMT比例偏低,启动时间偏晚,NEDA-3达标率低,治疗不达标时医生治疗策略调整存在惰性。

Objective To describe current status of diagnosis and treatment,clinical outcomes and treatment strategies of Chinese patients with multiple sclerosis(MS).Methods This study is a secondary data analysis of data collected by Adelphi MS Disease Specific Program(DSP),a cross-sectional survey conducted among neurologists(n=160)and their MS patients(n=956)in different district of China from June to August 2021.The endpoint of this study is the current status of diagnosis and treatment,clinical outcomes and treatment strategies.Results In preventive treatment,only 30.1%(287 cases)patients received the disease modifying treatment(DMT).The percentage of patients treated with DMT in relapsing-remitting MS(RRMS)[28.0%(215 cases)]was significantly lower than primary progressive MS(PPMS)[41.5%(34 cases)](P<0.05).Among all the patients treated with DMT,the median duration from diagnosis to receiving DMT was 2.5(1.0,5.4)years,and this duration in secondary progressive MS(SPMS)was the longest[9.0(4.3,11.8)years].Among 540 patients with complete follow-up data,27.4%patients achieved no evidence of disease activity(NEDA)-3 during the past 12 months.In addition,when patients had 1 relapse,disability progression without relapse,or active lesions in MRI without relapse,42.2%,39.1%and 62.1%doctors continued the current treatments,respectively.Conclusions In China,the percentages of MS patients achiving NEDA-3 were low.This might be due to the low percentage,the late initiation and conservative switching of DMT.