Abstract：
BACKGROUND: Numerous anti-seizure medications (ASMs) have been developed to treat Dravet syndrome (DS). This network meta-analysis aimed to comprehensively analyse the efficacy of ASMs in DS patients, especially in non-seizure-free patients after treatment.
METHODS: PubMed, EMBASE, Cochrane Library, and Chinese National Knowledge Infrastructure databases were searched. The treatment efficacy was assessed by the percentage reduction in monthly convulsive seizure frequency (MCSF) from baseline or individuals who achieved at least a 50 % or 75 % reduction from baseline in convulsive seizure frequency (CSF).
RESULTS: Six randomised controlled trials with 633 participants and seven regimens based on four add-on ASMs-fenfluramine (FFA), stiripentol (STP), cannabidiol (CBD), and soticlestat-were included. All drug regimens were superior to the placebo at achieving at least 50 % and 75 % reductions in CSF, but only STP, 0.4 mg/kg/d FFA (FFA0.4), and 0.7 mg/kg/d FFA (FFA0.7) reduced MCSF. STP (50 mg/kg/d) had the highest correlation with reducing MCSF and achieving at least a 50 % reduction from baseline in CSF, followed by FFA0.4 and FFA0.7. Soticlestat and CBD may also be effective in reducing seizures in DS patients.
CONCLUSIONS: STP can be recommended as the first choice among the included drug regimens for reducing seizures in DS patients, while FFA0.4 may be considered the second choice. Other drug regimens can be used as alternative treatments. STP, FFA0.4, and FFA0.7 may consistently present favourable efficacy in most DS patients, while other regimens may present prominent inter-individual variability. Appropriate dose selection and intense monitoring are necessary when treating DS using these drugs.
METHODS: PubMed, EMBASE, Cochrane Library, and Chinese National Knowledge Infrastructure databases were searched. The treatment efficacy was assessed by the percentage reduction in monthly convulsive seizure frequency (MCSF) from baseline or individuals who achieved at least a 50 % or 75 % reduction from baseline in convulsive seizure frequency (CSF).
RESULTS: Six randomised controlled trials with 633 participants and seven regimens based on four add-on ASMs-fenfluramine (FFA), stiripentol (STP), cannabidiol (CBD), and soticlestat-were included. All drug regimens were superior to the placebo at achieving at least 50 % and 75 % reductions in CSF, but only STP, 0.4 mg/kg/d FFA (FFA0.4), and 0.7 mg/kg/d FFA (FFA0.7) reduced MCSF. STP (50 mg/kg/d) had the highest correlation with reducing MCSF and achieving at least a 50 % reduction from baseline in CSF, followed by FFA0.4 and FFA0.7. Soticlestat and CBD may also be effective in reducing seizures in DS patients.
CONCLUSIONS: STP can be recommended as the first choice among the included drug regimens for reducing seizures in DS patients, while FFA0.4 may be considered the second choice. Other drug regimens can be used as alternative treatments. STP, FFA0.4, and FFA0.7 may consistently present favourable efficacy in most DS patients, while other regimens may present prominent inter-individual variability. Appropriate dose selection and intense monitoring are necessary when treating DS using these drugs.
摘要：
背景：已经开发了许多抗癫痫药物（ASM）来治疗Dravet综合征（DS）。本网络荟萃分析旨在全面分析ASM在DS患者中的疗效。尤其是治疗后未发作的患者。
方法：PubMed，EMBASE,科克伦图书馆,检索了中国国家知识基础设施数据库。通过每月惊厥性癫痫发作频率（MCSF）从基线或惊厥性癫痫发作频率（CSF）从基线降低至少50％或75％的个体减少来评估治疗功效。
结果：六个随机对照试验，包括633名参与者和七个方案，基于四个附加ASM-芬氟拉明（FFA），stiripentol（STP），大麻二酚（CBD），和seticlestat-被包括在内。所有药物方案都优于安慰剂，在CSF中至少减少了50％和75％，但只有STP,0.4mg/kg/dFFA（FFA0.4），和0.7mg/kg/dFFA（FFA0.7）降低MCSF。STP（50mg/kg/d）与减少MCSF并实现至少50％的相关性其次是FFA0.4和FFA0.7。Soticlestat和CBD也可以有效减少DS患者的癫痫发作。
结论：可以推荐STP作为纳入的减少DS患者癫痫发作的药物方案中的首选，而FFA0.4可能被认为是第二选择。其他药物方案可用作替代疗法。STP,FFA0.4和FFA0.7在大多数DS患者中可能始终表现出良好的疗效，而其他方案可能存在显著的个体间变异性。使用这些药物治疗DS时，需要适当的剂量选择和严格的监测。
方法：PubMed，EMBASE,科克伦图书馆,检索了中国国家知识基础设施数据库。通过每月惊厥性癫痫发作频率（MCSF）从基线或惊厥性癫痫发作频率（CSF）从基线降低至少50％或75％的个体减少来评估治疗功效。
结果：六个随机对照试验，包括633名参与者和七个方案，基于四个附加ASM-芬氟拉明（FFA），stiripentol（STP），大麻二酚（CBD），和seticlestat-被包括在内。所有药物方案都优于安慰剂，在CSF中至少减少了50％和75％，但只有STP,0.4mg/kg/dFFA（FFA0.4），和0.7mg/kg/dFFA（FFA0.7）降低MCSF。STP（50mg/kg/d）与减少MCSF并实现至少50％的相关性其次是FFA0.4和FFA0.7。Soticlestat和CBD也可以有效减少DS患者的癫痫发作。
结论：可以推荐STP作为纳入的减少DS患者癫痫发作的药物方案中的首选，而FFA0.4可能被认为是第二选择。其他药物方案可用作替代疗法。STP,FFA0.4和FFA0.7在大多数DS患者中可能始终表现出良好的疗效，而其他方案可能存在显著的个体间变异性。使用这些药物治疗DS时，需要适当的剂量选择和严格的监测。
