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
相关论文
共 50 条
  • [41] Safety of thiopental for the treatment of status epilepticus in Dravet syndrome
    Chiron, C.
    Nabbout, R.
    Chipaux, M.
    Desguerre, I
    Dulac, O.
    EPILEPSIA, 2007, 48 : 156 - 156
  • [42] Emerging drugs for the treatment of acromegaly
    Campana, Claudia
    Corica, Giuliana
    Nista, Federica
    Cocchiara, Francesco
    Graziani, Giulia
    Khorrami, Keyvan
    Franco, Marta
    Boschetti, Mara
    Ferone, Diego
    Gatto, Federico
    EXPERT OPINION ON EMERGING DRUGS, 2020, 25 (04) : 409 - 417
  • [43] Emerging drugs for the treatment of sepsis
    Heming, Nicholas
    Lamothe, Laure
    Ambrosi, Xavier
    Annane, Djillali
    EXPERT OPINION ON EMERGING DRUGS, 2016, 21 (01) : 27 - 37
  • [44] Emerging drugs for the treatment of onychomycosis
    Gupta, Aditya K.
    Stec, Nadia
    EXPERT OPINION ON EMERGING DRUGS, 2019, 24 (04) : 213 - 220
  • [45] Emerging drugs for the treatment of tuberculosis
    Yew, Wing Wai
    Cynamon, Michael
    Zhang, Ying
    EXPERT OPINION ON EMERGING DRUGS, 2011, 16 (01) : 1 - 21
  • [46] Emerging drugs for the treatment of myelofibrosis
    Geyer, Holly L.
    Mesa, Ruben A.
    EXPERT OPINION ON EMERGING DRUGS, 2015, 20 (04) : 663 - 678
  • [47] Emerging drugs for the treatment of hypercholesterolemia
    Bove, Marilisa
    Cicero, Arrigo F. G.
    Borghi, Claudio
    EXPERT OPINION ON EMERGING DRUGS, 2019, 24 (01) : 63 - 69
  • [48] Emerging drugs for the treatment of obesity
    Martinussen, Christoffer
    Bojsen-Moller, Kirstine Nyvold
    Svane, Maria Saur
    Dejgaard, Thomas Fremming
    Madsbad, Sten
    EXPERT OPINION ON EMERGING DRUGS, 2017, 22 (01) : 87 - 99
  • [49] Emerging drugs for the treatment of pruritus
    Stander, Sonja
    Weisshaar, Elke
    Raap, Ulrike
    EXPERT OPINION ON EMERGING DRUGS, 2015, 20 (03) : 515 - 521
  • [50] Emerging drugs for the treatment of uveitis
    Leung, Theresa G.
    Thorne, Jennifer E.
    EXPERT OPINION ON EMERGING DRUGS, 2013, 18 (04) : 513 - 521