Department of Children's Infectious Diseases, Medical University of Warsaw, Warsaw, Poland.
Department of Pediatric Infectious Diseases, Regional Hospital of Infectious Diseases in Warsaw, Warsaw, Poland.
Liver Int. 2023 Sep;43(9):1871-1878. doi: 10.1111/liv.15637. Epub 2023 Jun 8.
The aim of this non-commercial, open-label, real-life, non-randomized clinical trial was to analyse the efficacy and safety of a pangenotypic regimen sofosbuvir/velpatasvir (SOF/VEL) in patients aged 6-18 years with chronic hepatitis C virus (HCV) infection.
Fifty patients qualified for the 12-week treatment were divided into two weight groups: 15 children weighting between 17 and <30 kg received a fixed dose of 200/50 mg of SOF/VEL (tablet) once daily, and 35 patients weighting ≥30 kg were treated with 400/100 mg SOF/VEL. The primary endpoint of the study was efficacy defined as sustained viral response (undetectable HCV RNA using an real-time polymerase chain reaction method) at 12 weeks posttreatment (SVR12).
Median age of the participants was 10 (IQR 8-12) years, 47 were infected vertically, and 3 patients were previously ineffectively treated with pegylated interferon and ribavirin. Thirty-seven participants were infected with HCV genotype 1, 10 with HCV genotype 3 and the remaining 3 with genotype 4. There was no case of cirrhosis. SVR12 was 100%. Thirty-three reported adverse events (AEs) were considered related to the administration of SOF/VEL, all of them were mild or moderate. Children presenting with AEs were older compared to these without AEs: 12 (9.5-13) versus 9 (IQR 8-11) years (p = 0.008).
Results of the PANDAA-PED study indicated a 100% effectiveness of a 12-week therapy with SOF/VEL in children aged 6-18 years with chronic HCV infection and its good safety profile, in particular in younger patients.
本项非商业、开放标签、真实世界、非随机临床试验旨在分析泛基因型药物索磷布韦/维帕他韦（SOF/VEL）在慢性丙型肝炎病毒（HCV）感染 6-18 岁患者中的疗效和安全性。
符合 12 周治疗条件的 50 名患者分为两组体重：17-<30kg 的 15 名儿童每日一次服用固定剂量的 200/50mg SOF/VEL（片剂），35 名体重≥30kg 的患者接受 400/100mg SOF/VEL 治疗。研究的主要终点是治疗 12 周后（SVR12）定义的疗效，即持续病毒学应答（使用实时聚合酶链反应方法检测不到 HCV RNA）。
参与者的中位年龄为 10（IQR 8-12）岁，47 名参与者为垂直感染，3 名参与者既往接受聚乙二醇干扰素和利巴韦林治疗无效。37 名参与者感染 HCV 基因型 1，10 名感染 HCV 基因型 3，其余 3 名感染 HCV 基因型 4。无肝硬化病例。SVR12 为 100%。33 名参与者报告了 33 例（AE）与 SOF/VEL 给药相关的不良事件（AE），均为轻度或中度。发生 AE 的儿童年龄大于无 AE 的儿童：12（9.5-13）岁与 9（IQR 8-11）岁（p=0.008）。
PANDAA-PED 研究结果表明，SOF/VEL 12 周治疗在慢性 HCV 感染的 6-18 岁儿童中有效率为 100%，安全性良好，尤其是在年龄较小的患者中。
