UNASSIGNED: We searched PubMed, Embase (via Ovid), and Web of Science for relevant literature published before December 1st, 2023. Randomized, double-blind, placebo-controlled studies that evaluated the efficacy, safety, and tolerability of FFA in DS and LGS were identified and meta-analysis was performed according to doses. The study was registered with PROSPERO (CRD42023392454).
UNASSIGNED: Six hundred and twelve patients from four randomized controlled trials were enrolled. The results demonstrated that FFA at 0.2, 0.4, or 0.7 mg/kg/d showed significantly greater efficacy compared to placebo in terms of at least 50% reduction (p < 0.001, p < 0.001, p < 0.001) and at least 75% reduction (p < 0.001, p = 0.007, p < 0.001) in monthly seizure frequency from baseline. Moreover, significantly more patients receiving FFA than placebo were rated as much improved or very much improved in CGI-I by both caregivers/parents and investigators (p < 0.001). The most common treatment-emergent adverse events were decreased appetite, diarrhea, fatigue, and weight loss, with no valvular heart disease or pulmonary hypertension observed in any participant. For dose comparison, 0.7 mg/kg/d group presented higher efficacy on at least 75% reduction in seizure (p = 0.006) but not on at least 50% reduction. Weight loss (p = 0.002), decreased appetite (p = 0.04), and all-cause withdrawal (p = 0.036) were more common in 0.7 mg/kg/d group than 0.2 mg/kg/d. There was no statistical difference in other safety parameters between these two groups.
UNASSIGNED: The higher range of the licensed dose achieves the optimal balance between efficacy, safety, and tolerability in patients with DS and LGS.
UNASSIGNED: https://www.crd.york.ac.uk/PROSPERO/, identifier CRD42023392454.
■我们搜索了PubMed,Embase(通过Ovid),和WebofScience在12月1日之前发表的相关文献,2023年。随机化,双盲,评估疗效的安慰剂对照研究,安全,确定DS和LGS中FFA的耐受性,并根据剂量进行荟萃分析。该研究在PROSPERO(CRD42023392454)注册。
■纳入了来自四项随机对照试验的六百十二名患者。结果表明,与安慰剂相比,0.2、0.4或0.7mg/kg/d的FFA在从基线开始的每月发作频率中至少减少50%(p<0.001,p<0.001,p<0.001)和至少75%减少(p<0.001,p=0.007,p<0.001)方面显示出明显更大的功效。此外,护理人员/父母和研究者将接受FFA的患者的CGI-I评分为显著改善或非常改善(p<0.001).最常见的治疗引起的不良事件是食欲下降,腹泻,疲劳,和减肥,在任何参与者中均未观察到瓣膜性心脏病或肺动脉高压。对于剂量比较,0.7mg/kg/d组在癫痫发作减少至少75%(p=0.006)但没有减少至少50%时表现出更高的疗效。体重减轻(p=0.002),食欲下降(p=0.04),全因戒断(p=0.036)在0.7mg/kg/d组比0.2mg/kg/d组更常见。两组之间的其他安全性参数没有统计学差异。
■许可剂量的较高范围实现了功效之间的最佳平衡,安全,DS和LGS患者的耐受性。
■https://www.crd.约克。AC.英国/PROSPERO/,标识符CRD42023392454。