PT -期刊文章盟乔纳森·哈尔福德AU -英格丽德雅伯盟裂缝Nabbout AU - Rocio Sanchez-Carpintero盟雅艾尔希洛Malawsky AU -马修王盟-丹尼尔Checketts盟Kevan gosper VanLandingham TI -附加的长期安全性和有效性大麻二酚(CBD)治疗Dravet综合症患者在一个开放的扩展试验(p5.5 - 005) DP - 2019年4月09年TA -神经病学第六PG - p5.5 - 005 - 92 IP - 15补充4099 - //www.ez-admanager.com/content/92/15_supplement/p5.5 005. - 005.短4100 - //www.ez-admanager.com/content/92/15_supplement/p首页5.5 - -全所以Neurology2019 4月09年;92 AB -目的:评估附加CBD的长期安全性和有效性治疗Dravet综合症患者(DS)在第二个临时开放的扩展(OLE)的分析两个阶段3,随机对照试验,GWPCARE1 GWPCARE2。背景:DS是一种难治性癫痫,发作通常在生命的第一年和高死亡率。高纯度CBD (Epidiolex®)批准在美国癫痫与Dravet综合征患者中≥2岁。设计/方法:患者接受吉瓦的高纯度植物制药配方CBD(100毫克/毫升)在口腔的解决方案。主要终点是安全。二级端点从基线变化剧烈和总发作频率和主题/照顾者全球变化的印象。结果:289名患者完成了随机对照试验,278年GWPCARE5登记。在14%的病人已完成数据截止,52%是持续的,和34%的退出了。意味着病人年龄是9.8岁;97%的人& lt; 18年。患者服用平均3并发抗癫痫药物有68% clobazam, stiripentol丙戊酸钠,63%和39%。 The mean of patients’ modal CBD dose was 22 mg/kg/d. Adverse events (AEs) and serious AEs were reported by 96% and 32% of patients; 7% discontinued due to AEs. Elevated liver transaminases >3× upper limit of normal were reported in 9% of patients; none had severe liver injury. Two nontreatment-related deaths were reported. Median % reductions from baseline assessed in 12-week intervals were 44%–57% for convulsive and 49%–67% for total seizures through 72 weeks. Over 80% of patients/caregivers reported improvements in overall condition.Conclusions: Long-term treatment with add-on CBD in patients with DS produced sustained seizure reductions, with no new safety concerns.Disclosure: Dr. Halford has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Brain Sentinel, Greenwich Biosciences, Philips Neuro, NCGS. Dr. Halford has received research support from Biogen, Greenwich Biosciences, Optima Neurosciences, SK Life Sciences, Takeda, Brain Sentinel, Philips, and the Epilepsy Study Consortium. Dr. Scheffer has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with BioMarin and Xenon. Dr. Nabbout has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Novartis, Livanova, Advicennes, GW Pharma, Zogenix, UCB, Eisai, Biomarin, Targeon, Takeda, Nutricia, Eisai, and Zogenix. Dr. Nabbout has received personal compensation in an editorial capacity for Epilepsia and Epilepsia open. Dr. Sanchez-Carpintero has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with advisory board for Novartis Pharmaceuticals and GWPharma. Dr. Sanchez-Carpintero has received research support from GWpharma and Zogenix. Dr. Shiloh-Malawsky has nothing to disclose. Dr. Wong has received research support from GW Pharma. Dr. Checketts has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with GW Research Ltd. Dr. Checketts holds stock and/or stock options in GW Pharmaceuticals. Dr. VanLandingham has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Greenwich Biosciences Inc.