Mizuochi Tatsuki, Abukawa Daiki, Inui Ayano, Azuma Yoshihiro, Suzuki Takako, Yagi Hiroko, Kumagai Hideki, Mushiake Sotaro, Tokuhara Daisuke, Tsumura Naoya, Kato Ken, Tanaka Yasuhito, Tajiri Hitoshi
Department of Pediatrics and Child Health, Kurume University School of Medicine, Kurume, Japan.
Department of Gastroenterology and Hepatology, Miyagi Children's Hospital, Sendai, Japan.
Hepatol Res. 2025 Jul 21. doi: 10.1111/hepr.70004.
Part 2 of the DORA study, an international clinical trial evaluating glecaprevir and pibrentasvir (G/P) treatment in children aged 3-11 years with chronic hepatitis C virus (HCV) infection, demonstrated high efficacy and safety. However, there is limited evidence regarding real-world use of G/P in this pediatric population. This prospective multicenter study in Japan evaluated the real-world efficacy and safety of G/P treatment in children aged 3-11 years with chronic HCV.
Children aged 3-11 years with chronic HCV were prospectively enrolled and received a once-daily dose of G/P for either 8 or 12 weeks. The primary endpoint was sustained virologic response at 12 weeks after treatment completion (SVR12). Safety was assessed through adverse events, laboratory tests, and growth measurements.
A total of 18 children (8 girls) from 9 pediatric centers in Japan were enrolled, with a median age of 9 years (range, 3-11). Genotype distribution was as follows: 1b (n = 3), 2a (n = 8), 2b (n = 5), 3a (n = 1), and unknown of Serotype 2 (n = 1). All participants were treatment-naïve and completed G/P treatment (17 for 8 weeks, 1 for 12 weeks). SVR12 was achieved in 17 patients (94%). Most adverse events were mild, with no serious events. Treatment led to significant reductions in serum alanine aminotransferase and Wisteria floribunda agglutinin-positive Mac-2 binding protein levels. No impairments in growth were observed.
In real-world clinical practice, G/P treatment demonstrated high efficacy and good tolerability in children aged 3-11 years with chronic HCV.
DORA研究的第二部分是一项国际临床试验，评估glecaprevir和pibrentasvir（G/P）对3至11岁慢性丙型肝炎病毒（HCV）感染儿童的治疗效果，结果显示其具有高疗效和安全性。然而，关于G/P在该儿科人群中的实际应用证据有限。这项在日本进行的前瞻性多中心研究评估了G/P治疗3至11岁慢性HCV儿童的实际疗效和安全性。
前瞻性纳入3至11岁的慢性HCV儿童，每日服用一次G/P，疗程为8周或12周。主要终点是治疗完成后12周的持续病毒学应答（SVR12）。通过不良事件、实验室检查和生长测量评估安全性。
日本9个儿科中心共纳入18名儿童（8名女孩），中位年龄为9岁（范围3至11岁）。基因型分布如下：1b型（n = 3）、2a型（n = 8）、2b型（n = 5）、3a型（n = 1）和2型血清型未知（n = 1）。所有参与者均未接受过治疗且完成了G/P治疗（17例接受8周治疗，1例接受12周治疗）。17例患者（94%）实现了SVR12。大多数不良事件为轻度，无严重事件。治疗导致血清丙氨酸氨基转移酶和紫藤凝集素阳性Mac-2结合蛋白水平显著降低。未观察到生长发育受损情况。
在实际临床实践中，G/P治疗对3至11岁慢性HCV儿童显示出高疗效和良好的耐受性。
