Assistance Publique-Hôpitaux de Paris (APHP), Member of the European Reference Network (ERN) EpiCARE, Necker Enfants Malades University Hospital, Université Paris Cité, 149 Rue de Sèvres, 75015 Paris, France; INSERM U1163, Institut Imagine, 24 Bd du Montparnasse, 75015 Paris, France.
Paediatric Epilepsy Department, Coordinator of the ERN EpiCARE, University Hospitals of Lyon, 59 Bd Pinel, 60700 Lyon, France; Epilepsy Research Unit, Member of the ERN EpiCARE, Hospital San Juan de Déu, Passeig de Sant Joan de Déu, 2, 08950 Esplugues de Llobregat, Barcelona, Spain.
Seizure. 2023 Aug;110:78-85. doi: 10.1016/j.seizure.2023.05.003. Epub 2023 May 5.
This retrospective chart review study (GWEP20052) evaluated plant-derived highly purified cannabidiol (CBD; Epidyolex®; 100 mg/mL oral solution) use without clobazam as add-on therapy in patients aged ≥2 years with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) enrolled in a European Early Access Program.
Data were extracted from patient charts covering a period starting 3 months before CBD treatment and concluding after 12 months of CBD treatment, or sooner if a patient discontinued CBD or started clobazam.
Of 114 enrolled patients, data were available for 107 (92 LGS, 15 DS) who received CBD without clobazam for ≥3 months. Mean age: 14.5 (LGS) and 10.5 (DS) years; female: 44% (LGS) and 67% (DS). Mean time-averaged CBD dose: 13.54 (LGS) and 11.56 (DS) mg/kg/day. Median change from baseline in seizure frequency per 28 days over 3-month intervals varied from -6.2% to -20.9% for LGS and 0% to -16.7% for DS. Achievement of ≥50% reduction in drop (LGS) or convulsive (DS) seizures at 3 and 12 months: LGS, 19% (n = 69) and 30% (n = 53); DS, 21% (n = 14) and 13% (n = 8). Retention on CBD without clobazam (enrolled set): 94%, 80%, 69%, and 63% at 3, 6, 9, and 12 months. Adverse event (AE) incidence was 31%, most commonly somnolence, seizure, diarrhea, and decreased appetite. Two patients discontinued CBD owing to AEs, and four patients with LGS experienced elevated liver enzymes.
Results support favorable effectiveness and retention of CBD without concomitant clobazam for up to 12 months in clinical practice.
本回顾性图表研究（GWEP20052）评估了植物来源的高纯度大麻二酚（CBD；Epidyolex®；100mg/mL 口服溶液）在未加用氯巴占的情况下作为附加疗法，用于年龄≥2 岁的 Lennox-Gastaut 综合征（LGS）或 Dravet 综合征（DS）患者。这些患者入组了一项欧洲早期准入计划。
从患者图表中提取数据，数据覆盖了开始使用 CBD 治疗前 3 个月到治疗后 12 个月的时间段，或者如果患者停止使用 CBD 或开始使用氯巴占，则以更早的时间点为准。
在 114 名入组患者中，107 名（92 名 LGS，15 名 DS）患者接受了≥3 个月未加用氯巴占的 CBD 治疗，数据可用于分析。患者平均年龄：14.5 岁（LGS）和 10.5 岁（DS）；女性：44%（LGS）和 67%（DS）。平均时间加权 CBD 剂量：13.54mg/kg/天（LGS）和 11.56mg/kg/天（DS）。3 个月和 12 个月期间，每 28 天发作频率的中位数变化范围为 LGS 从基线下降 6.2%至下降 20.9%，DS 为 0%至下降 16.7%。3 个月和 12 个月时，≥50%减少癫痫发作（LGS）或癫痫发作（DS）的患者比例：LGS，19%（n=69）和 30%（n=53）；DS，21%（n=14）和 13%（n=8）。未加用氯巴占的 CBD 保留率（入组人群）：3 个月时为 94%，6 个月时为 80%，9 个月时为 69%，12 个月时为 63%。不良事件（AE）发生率为 31%，最常见的是嗜睡、癫痫发作、腹泻和食欲下降。两名患者因 AE 停用 CBD，四名 LGS 患者出现肝酶升高。
结果支持在临床实践中，CBD 不加用氯巴占，最长可使用 12 个月，具有良好的有效性和保留率。
