Emerging drugs for the treatment of Dravet syndrome

被引:25
|
作者
Brigo, Francesco [1 ,2 ]
Striano, Pasquale [3 ]
Balagura, Ganna [3 ]
Belcastro, Vincenzo [4 ]
机构
[1] Univ Verona, Dept Neurosci Biomed & Movement Sci, Verona, Italy
[2] Franz Tappeiner Hosp, Div Neurol, Merano, Italy
[3] Univ Genoa, G Gaslini Inst, Dept Neurosci Rehabil Ophthalmol Genet Maternal &, Pediat Neurol & Muscular Dis Unit, Genoa, Italy
[4] St Anna Hosp, Neurol Unit, Como, Italy
关键词
Cannabidiol; Dravet syndrome; fenfluramine; lorcaserin; trazodone; verapamil; SEVERE MYOCLONIC EPILEPSY; LOW-DOSE FENFLURAMINE; ANTIEPILEPTIC DRUG; KETOGENIC DIET; VERAPAMIL; EFFICACY; SEIZURES; CANNABIDIOL; STIRIPENTOL; COMORBIDITIES;
D O I
10.1080/14728214.2018.1552937
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Introduction: Dravet syndrome (DS) is an early-onset genetic developmental epileptic encephalopathy characterized by multiple seizure types which are refractory to antiseizure medication. There is an unmet need for effective and tolerable drugs to control different seizure types in DS types, with the aim of improving quality of life and preventing neurological impairment. Areas covered: Narrative review of efficacy and tolerability of fenfluramine, cannabidiol (CBD), verapamil, and modulators of serotonin signaling pathways (lorcaserin or trazodone) in the treatment of DS. Expert opinion/commentary: A recent large randomized controlledtrial has shown that CBD is effective in the treatment of DS; preliminary data from the placebo-controlled trial on fenfluramine are also promising. Further studies are definitely required to evaluate the role of verapamil and modulators of serotonin signaling in DS. At present, drugs used to treat seizures in DS treat the symptoms of epilepsy rather than its cause(s). Future research should focus on elucidating the natural history of DS and whether appropriate treatment can have a beneficial impact on its disease course. A multidisciplinary, individualized approach to care of DS patients is required.
引用
收藏
页码:261 / 269
页数:9
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