Chowdhury Selia, Chowdhury Samia
Dhaka Medical College, Dhaka, Bangladesh.
Sylhet MAG Osmani Medical College, Sylhet, Bangladesh.
Discoveries (Craiova). 2025 Jun 30;13(1):e208. doi: 10.15190/d.2025.7. eCollection 2025 Apr-Jun.
Ganaxolone, a neuroactive steroid that increases GABAergic inhibition, has been tested in many trials for the resolution of different epileptic disorders. Based on these, our study implemented a systematic review to evaluate the general benefit of ganaxolone for seizures. Pubmed, google scholar, European PubMed Central, Cochrane Library, and U.S. National Library of Medicine Clinical Trials were searched for relevant randomized controlled trials (RCTs) up to March 2023 and 12 RCTs were included in this review. We included 1337 patients from 12 RCTs to evaluate the efficacy and safety of ganaxolone. As results showed, across all trials, 26.3% of participants reported >50% reduction from baseline in seizure frequency. The analysis revealed that the nervous system was the most impacted, with dizziness, somnolence, headache, and seizures being the most frequently reported treatment-related adverse events (TRAEs). The results of these trials suggest that ganaxolone may be effective in reducing seizure frequency in patients with various types of epilepsy, including CDKL5 deficiency disorder, Lennox-Gastaut syndrome, and Protocadherin 19. However, further studies are needed to determine its safety and efficacy in a larger patient population.
甘氨酰环己酮，一种可增强γ-氨基丁酸（GABA）能抑制作用的神经活性甾体，已在许多试验中用于治疗不同类型的癫痫疾病。基于这些研究，我们的研究进行了一项系统评价，以评估甘氨酰环己酮对癫痫发作的总体疗效。检索了PubMed、谷歌学术、欧洲PubMed中心、Cochrane图书馆和美国国立医学图书馆临床试验数据库，查找截至2023年3月的相关随机对照试验（RCT），本评价纳入了12项RCT。我们纳入了来自12项RCT的1337例患者，以评估甘氨酰环己酮的疗效和安全性。结果显示，在所有试验中，26.3%的参与者报告癫痫发作频率较基线降低超过50%。分析表明，神经系统受影响最大，头晕、嗜睡、头痛和癫痫发作是最常报告的与治疗相关的不良事件（TRAEs）。这些试验结果表明，甘氨酰环己酮可能对降低包括CDKL5缺乏症、Lennox-Gastaut综合征和原钙黏蛋白19在内的各种类型癫痫患者的癫痫发作频率有效。然而，需要进一步研究以确定其在更大患者群体中的安全性和疗效。
