Desnous Béatrice, Beretti Thibault, Muller Nathan, Neveu Julien, Villeneuve Nathalie, Lépine Anne, Daquin Géraldine, Milh Mathieu
Pediatric Neurology Department, Timone Enfant, APHM, Marseille, France.
Pediatric Neurology Department, Lenval, Nice, France.
Epilepsia Open. 2024 Feb;9(1):397-403. doi: 10.1002/epi4.12796. Epub 2023 Nov 15.
We aim to assess the efficacy and tolerance of cannabidiol as adjunctive therapy for Rett syndrome (RTT) patients with epilepsy. We conducted a longitudinal observational study through a monocentric cohort of 46 patients with RTT. Patients were recruited from March 2020 to October 2022 and were treated with Epidyolex® (cannabidiol, CBD, 100 mg/mL oral solution). In our cohort, 26 patients had associated epilepsy (26/46 [56%]), and 10/26 (38%) were treated with CBD, in combination with clobazam in 50% of cases. The median dose at their last follow-up was 15 mg/kg/day. The median treatment duration was 13 months (range: 1-32 months). CBD reduced the incidence of seizures in seven out of 10 patients (70%) with one seizure-free patient, two patients with a reduction of seizures of more than 75%, and four patients with a decrease of more than 50%. No aggravation of symptoms or adverse effects were observed. Only one patient experienced a transitory drooling and somnolence episode at the CBD initiation. Half of the patients showed a reduction in agitation and/or anxiety attacks, and an improvement in spasticity was reported in 4/10 (40%) of patients. CBD appears to have potential therapeutic value for the treatment of drug-resistant epilepsy in Rett syndrome. CBD is well tolerated and, when used in combination with clobazam, may increase the effectiveness of clobazam alone.
我们旨在评估大麻二酚作为雷特综合征（RTT）癫痫患者辅助治疗的疗效和耐受性。我们通过一个单中心队列的46例RTT患者进行了一项纵向观察性研究。患者于2020年3月至2022年10月招募，并接受Epidyolex®（大麻二酚，CBD，100mg/mL口服溶液）治疗。在我们的队列中，26例患者伴有癫痫（26/46 [56%]），其中10/26（38%）接受了CBD治疗，50%的病例与氯巴占联合使用。他们最后一次随访时的中位剂量为15mg/kg/天。中位治疗持续时间为13个月（范围：1 - 32个月）。CBD使10例患者中的7例（70%）癫痫发作次数减少，其中1例无癫痫发作，2例癫痫发作减少超过75%，4例减少超过50%。未观察到症状加重或不良反应。只有1例患者在开始使用CBD时出现短暂的流口水和嗜睡发作。一半的患者躁动和/或焦虑发作减少，4/10（40%）的患者报告痉挛有所改善。大麻二酚似乎对雷特综合征耐药性癫痫的治疗具有潜在的治疗价值。大麻二酚耐受性良好，与氯巴占联合使用时，可能会提高氯巴占单独使用的有效性。
