特立氟胺和西尼莫德对复发型多发性硬化的有效性和安全性

Efficacy and Safety of Terfluramide and Siponimod in the Treatment of Relapsing Multiple Sclerosis

目的 探讨口服特立氟胺或西尼莫德对复发型多发性硬化(RMS)患者的有效性和安全性。方法 本研究是一项单中心回顾性观察性研究,纳入于郑州大学第一附属医院多发性硬化中心确诊为RMS、连续服用特立氟胺(14 mg·d^(-1))或西尼莫德(2 mg·d^(-1))至少6个月的患者。本研究采用年复发率(ARR)、中枢神经系统MRI T_(2)FLAIR序列病灶数评估疾病活动度,采用扩展残疾量表评分(EDSS)评估患者残疾功能。记录患者使用特立氟胺或西尼莫德治疗6个月后的复发和残疾进展情况,并对比两组的疗效和安全性。主要观察指标为使用特立氟胺或西尼莫德治疗至少6个月后的ARR、治疗12个月时的疾病无复发率;其他指标为使用特立氟胺或西尼莫德治疗至少6个月后患者的MRI T_(2)FLAIR序列病灶数目、EDSS评分、不良反应及用药中断情况,所有数据使用SPSS 26.0统计软件进行处理。结果 最终特立氟胺组纳入25例患者,西尼莫德组纳入28例患者。患者口服特立氟胺的用药时间为16(6~20)个月,用药6个月后ARR、EDSS评分较用药前下降(P<0.05),25%患者的MRI T_(2)FLAIR序列病灶数增加,用药12个月无复发率为62.50%。患者口服西尼莫德的用药时间为15(6~20)个月,用药6个月后ARR评分较用药前下降(P<0.05),18.18%患者的MRI T_(2)FLAIR序列病灶数增加,用药12个月无复发率为75.00%。特立氟胺组6例患者出现不良反应,最常见的是脱发;西尼莫德组12例患者出现不良反应,最常见的是肝酶异常,其中因肝衰竭暂停西尼莫德治疗1例。结论 在本次单中心观察研究中,特立氟胺和西尼莫德均能有效降低RMS患者的ARR,其中西尼莫德较特立氟胺疗效更好,但安全性较差。

Objective To explore the efficacy and safety of oral terfluramide and siponimod in patients with relapsing multiple sclerosis(RMS).Methods This study was a single center retrospective observational study,and patients who were diagnosed as RMS at the Multiple Sclerosis Center of the First Affiliated Hospital of Zhengzhou University and had been taking terfluramide(14 mg·d-1)or siponimod(2 mg·d-1)continuously for at least 6 months were selected.The annualized relapse rate(ARR)and the number of central nervous system MRI T_(2)FLAIR lesions were used to assess the degree of disease activity,the expanded disability status scale(EDSS)was used to evaluate the patients’disability status.The relapse and disability progression were recorded after 6 months of treatment with terifluramide or siponimod,and the effectiveness and safety of the two groups were compared.The main observed indicators were ARR after initiation of terifluramide or siponimod treatment for at least 6 months and the proportion of relapse freedom after 12 months of treatment.The other observed indicators were the number of MRI T_(2)FLAIR lesions,EDSS scores,adverse effects and drug discontinuation in patients at least 6 months after initiation of teriflunomide or siponimod.All data were processed by SPSS 26.0 statistical software.Results Finally,25 patients were included in the terifluramide group and 28 patients were included in the siponimod group.The duration of oral terifluramide was 16(6-20)months,and the ARR and the EDSS score after 6 months of treatment were lower than before treatment(P<0.05),the number of MRI T_(2)FLAIR lesions increased in 25%of the patients,and the relapse freedom rate after 12 months of medication was 62.50%.The duration of oral siponimod was 15(6-20)months,and the ARR score after 6 months of treatment was lower than before treatment(P<0.05),the number of MRI T_(2)FLAIR lesions increased in 18.18%of the patients,and the relapse freedom rate after 12 months of medication was 75.00%.Six patients in the tertifluramide group experienced adverse reactions,the most common being hair loss.In the siponimod group,12 patients experienced adverse reactions,the most common of which was abnormal liver enzymes.Among them,1 patient was suspended from siponimod treatment due to liver failure.Conclusion In this single center observational study,both terifluramide and siponimod can effectively reduce the ARR of patients,and siponimod has better efficacy and poor safety than terifluramide.