Takeda Mutsuki, Kitazawa Yu, Sonoda Masaki, Morihara Keisuke, Miyaji Yosuke, Higashiyama Yuichi, Kimura Katsuo, Ueda Naohisa, Doi Hiroshi, Tanaka Fumiaki
Department of Neurology and Stroke Medicine, Yokohama City University Graduate School of Medicine, Yokohama, JPN.
Department of Neurosurgery, Yokohama City University Graduate School of Medicine, Yokohama, JPN.
Cureus. 2025 Jul 3;17(7):e87250. doi: 10.7759/cureus.87250. eCollection 2025 Jul.
Purpose Fenfluramine has received approval for treating seizures in individuals with Lennox-Gastaut syndrome (LGS). Nonetheless, Phase III trials mainly focused on children, leaving limited information regarding its effectiveness and safety in adults. This case series aimed to elucidate these factors in adult patients. Methods This case series examined the clinical progression of adult patients diagnosed with LGS who received fenfluramine at our clinic following its approval in Japan in March 2024, extending through February 2025. Fenfluramine was administered to those experiencing drop attacks and generalized slow spike-and-wave complexes, fulfilling the conventional diagnostic criteria for LGS, and their responses to treatment were analyzed. Results This case series involved nine adult patients aged 18-49. All patients demonstrated efficacy, with seven (78%) experiencing at least a 50% reduction in seizure frequency. No one needed maintenance doses above the recommended starting minimum of 0.2 mg/kg/day, and five patients (56%) achieved seizure control with even lower doses. Remarkably, three patients who did not meet the latest 2022 diagnostic criteria also demonstrated positive responses. Adverse events were reported in five patients (56%), primarily including somnolence, diarrhea, and anorexia. In these situations, a dose reduction to half was necessary, yet efficacy was maintained. Conclusion Low-dose fenfluramine may effectively manage seizures in adult patients with LGS while reducing adverse effects. This case series provides a growing body of real-world data regarding the use of fenfluramine in this patient population.
目的 芬氟拉明已获批用于治疗 Lennox-Gastaut 综合征(LGS)患者的癫痫发作。尽管如此,III 期试验主要针对儿童,关于其在成人中的有效性和安全性的信息有限。本病例系列旨在阐明成年患者中的这些因素。方法 本病例系列研究了 2024 年 3 月在日本获批后,于我们诊所接受芬氟拉明治疗的成年 LGS 患者的临床进展,时间跨度至 2025 年 2 月。对那些经历跌倒发作和广泛性慢棘慢复合波、符合 LGS 传统诊断标准的患者给予芬氟拉明,并分析他们的治疗反应。结果 本病例系列包括 9 名年龄在 18 - 49 岁的成年患者。所有患者均显示出疗效,7 名(78%)患者的癫痫发作频率至少降低了 50%。没有人需要高于推荐起始最小剂量 0.2 mg/kg/天的维持剂量,5 名患者(56%)使用更低剂量就实现了癫痫控制。值得注意的是,3 名不符合 2022 年最新诊断标准的患者也显示出阳性反应。5 名患者(56%)报告了不良事件,主要包括嗜睡、腹泻和厌食。在这些情况下,有必要将剂量减半,但疗效得以维持。结论 低剂量芬氟拉明可能有效控制成年 LGS 患者的癫痫发作,同时减少不良反应。本病例系列提供了越来越多关于芬氟拉明在该患者群体中使用的真实世界数据。