Dalfampridine in hereditary spastic paraplegia: a prospective, open study

被引：17

作者：

Bereau, Matthieu ^{[1
]}

Anheim, Mathieu ^{[2
,3
,4
]}

Chanson, Jean-Baptiste ^{[2
]}

Tio, Gregory ^{[5
]}

Echaniz-Laguna, Andoni ^{[2
]}

Depienne, Christel ^{[6
]}

Collongues, Nicolas ^{[2
,7
]}

de Seze, Jerome ^{[2
,7
]}

机构：

[1] CHRU Besancon, Dept Neurol, Besancon, France

[2] Hospices Civils Strasbourg, Dept Neurol, F-67091 Strasbourg, France

[3] Univ Strasbourg, FMTS, Strasbourg, France

[4] Univ Strasbourg, IGBMC, INSERM, U964,CNRS,UMR7104, F-67404 Illkirch Graffenstaden, France

[5] CHRU Besancon, Dept Stat, Ctr Invest Clin & Innovat Technol, INSERM,CIT 808, Besancon, France

[6] Grp Hosp Pitie Salpetriere, Dept Genet Cytogenet & Embryol, F-75634 Paris, France

[7] Hospices Civils Strasbourg, Ctr Invest Clin Plurithemat, INSERM, CIC P1002, Strasbourg, France

来源：

JOURNAL OF NEUROLOGY | 2015年 / 262卷 / 05期

关键词：

Hereditary spastic paraplegia; Genetics; Pharmacology; Gait; Walking ability; Clinical trial; SPINAL-CORD-INJURY; MULTIPLE-SCLEROSIS; DOUBLE-BLIND; TRIAL; 4-AMINOPYRIDINE; PARAPARESIS; FAMPRIDINE;

D O I：

10.1007/s00415-015-7707-6

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

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.

引用

页码：1285 / 1288

页数：4

共 13 条

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