{"title":"标签外使用大麻二酚治疗遗传性癫痫和发育性脑病：病例报告","authors":"Elisa Mannini , Francesco Misirocchi , Stefania Lazzari , Giulia Balella , Dario Bottignole , Maddalena Frapporti , Lucia Zinno , Irene Florindo , Liborio Parrino , Carlotta Mutti","doi":"10.1016/j.ebr.2024.100687","DOIUrl":null,"url":null,"abstract":"<div><p>Developmental Epileptic encephalopathies (DEEs) are severe neurological conditions where cognitive functions appear modulated by both seizure and interictal epileptiform activity. Cannabidiol (CBD) has been shown to be highly effective in the treatment of drug-resistant seizures in patients with DEEs. Along with its antiseizure effects, CBD demonstrated clinical beneficial effects in patients’ quality of life, sleep and numerous adaptive behaviors. However, based on the available phase III studies, the indications for this treatment have so far been restricted to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS) and tuberous sclerosis complex (TSC) by regulatory authorities. We present the case of a 30-year-old girl with a rare genetic DEE, experiencing relevant seizure frequency reduction together with striking improvement in sleep quality, mood, behavior, language and motor skills after introducing off-label CBD.</p></div>","PeriodicalId":36558,"journal":{"name":"Epilepsy and Behavior Reports","volume":"27 ","pages":"Article 100687"},"PeriodicalIF":1.8000,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2589986424000443/pdfft?md5=fd3325f0c7ff8b1d3fa9b7438c288400&pid=1-s2.0-S2589986424000443-main.pdf","citationCount":"0","resultStr":"{\"title\":\"Off-label use of cannabidiol in genetic epileptic and developmental encephalopathies: A case report\",\"authors\":\"Elisa Mannini , Francesco Misirocchi , Stefania Lazzari , Giulia Balella , Dario Bottignole , Maddalena Frapporti , Lucia Zinno , Irene Florindo , Liborio Parrino , Carlotta Mutti\",\"doi\":\"10.1016/j.ebr.2024.100687\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><p>Developmental Epileptic encephalopathies (DEEs) are severe neurological conditions where cognitive functions appear modulated by both seizure and interictal epileptiform activity. Cannabidiol (CBD) has been shown to be highly effective in the treatment of drug-resistant seizures in patients with DEEs. Along with its antiseizure effects, CBD demonstrated clinical beneficial effects in patients’ quality of life, sleep and numerous adaptive behaviors. However, based on the available phase III studies, the indications for this treatment have so far been restricted to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS) and tuberous sclerosis complex (TSC) by regulatory authorities. We present the case of a 30-year-old girl with a rare genetic DEE, experiencing relevant seizure frequency reduction together with striking improvement in sleep quality, mood, behavior, language and motor skills after introducing off-label CBD.</p></div>\",\"PeriodicalId\":36558,\"journal\":{\"name\":\"Epilepsy and Behavior Reports\",\"volume\":\"27 \",\"pages\":\"Article 100687\"},\"PeriodicalIF\":1.8000,\"publicationDate\":\"2024-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.sciencedirect.com/science/article/pii/S2589986424000443/pdfft?md5=fd3325f0c7ff8b1d3fa9b7438c288400&pid=1-s2.0-S2589986424000443-main.pdf\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Epilepsy and Behavior Reports\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S2589986424000443\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Epilepsy and Behavior Reports","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2589986424000443","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
引用次数: 0
摘要
发育性癫痫性脑病（DEEs）是一种严重的神经系统疾病，其认知功能受到癫痫发作和发作间期癫痫样活动的影响。研究表明，大麻二酚（CBD）对治疗 DEEs 患者的耐药性癫痫发作非常有效。除了抗癫痫作用外，大麻二酚还对患者的生活质量、睡眠和多种适应行为有临床益处。然而，根据现有的 III 期研究，迄今为止，监管机构将这种治疗方法的适应症限制在伦诺克斯-加斯豪特综合征（LGS）、德拉沃综合征（DS）和结节性硬化综合征（TSC）。我们介绍了一个患有罕见遗传性 DEE 的 30 岁女孩的病例，她在标示外使用 CBD 治疗后，癫痫发作频率明显降低，睡眠质量、情绪、行为、语言和运动技能也显著改善。
Off-label use of cannabidiol in genetic epileptic and developmental encephalopathies: A case report
Developmental Epileptic encephalopathies (DEEs) are severe neurological conditions where cognitive functions appear modulated by both seizure and interictal epileptiform activity. Cannabidiol (CBD) has been shown to be highly effective in the treatment of drug-resistant seizures in patients with DEEs. Along with its antiseizure effects, CBD demonstrated clinical beneficial effects in patients’ quality of life, sleep and numerous adaptive behaviors. However, based on the available phase III studies, the indications for this treatment have so far been restricted to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS) and tuberous sclerosis complex (TSC) by regulatory authorities. We present the case of a 30-year-old girl with a rare genetic DEE, experiencing relevant seizure frequency reduction together with striking improvement in sleep quality, mood, behavior, language and motor skills after introducing off-label CBD.
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