Béreau Matthieu, Anheim Mathieu, Chanson Jean-Baptiste, Tio Grégory, Echaniz-Laguna Andoni, Depienne Christel, Collongues Nicolas, de Sèze Jérôme
Département de Neurologie, CHRU de Besançon, Besançon, France.
J Neurol. 2015 May;262(5):1285-8. doi: 10.1007/s00415-015-7707-6. Epub 2015 Mar 26.
Our aim was to support the use of dalfampridine as a treatment for patients affected with hereditary spastic paraplegia (HSP). We performed a prospective, uncontrolled, proof of concept, open trial. We included 12 HSP patients defining the total group (TG) who received dalfampridine 10 mg twice daily for 2 weeks. Efficacy assessment was based on walking ability improvement. The Timed-25-Foot Walk Test, the Spastic Paraplegia Rating Scale (SPRS), and the 12-item Multiple Sclerosis Walking Scale (MSWS-12) were performed before and after treatment. Safety assessment was based on adverse events occurrence. A significant improvement in SPRS (p = 0.0195) and MSWS-12 (p = 0.0429) was noted after treatment in the TG. No serious adverse events were noted. This interventional study provides encouraging results supporting the use of dalfampridine in HSP.
我们的目的是支持将达氟吡啶用作治疗遗传性痉挛性截瘫(HSP)患者的药物。我们开展了一项前瞻性、非对照、概念验证性开放试验。我们纳入了12名HSP患者组成总组(TG),这些患者接受每日两次、每次10毫克的达氟吡啶治疗,为期2周。疗效评估基于步行能力的改善情况。在治疗前后分别进行了25英尺计时步行测试、痉挛性截瘫评定量表(SPRS)和12项多发性硬化步行量表(MSWS-12)评估。安全性评估基于不良事件的发生情况。总组患者在治疗后SPRS(p = 0.0195)和MSWS-12(p = 0.0429)有显著改善。未观察到严重不良事件。这项干预性研究提供了令人鼓舞的结果,支持在HSP中使用达氟吡啶。