Mizuochi Tatsuki, Iwama Itaru, Inui Ayano, Ito Yoshinori, Takaki Yugo, Mushiake Sotaro, Tokuhara Daisuke, Ishige Takashi, Ito Koichi, Murakami Jun, Hishiki Haruka, Mikami Hitoshi, Bessho Kazuhiko, Kato Ken, Yasuda Ryosuke, Yamashita Yushiro, Tanaka Yasuhito, Tajiri Hitoshi
Department of Pediatrics and Child Health, Kurume University School of Medicine, 67 Asahi-machi, Kurume, 8300011, Japan.
Division of Gastroenterology and Hepatology, Saitama Children's Medical Center, Saitama, Japan.
J Gastroenterol. 2023 Apr;58(4):405-412. doi: 10.1007/s00535-023-01968-x. Epub 2023 Feb 15.
Part 1 of the DORA study, a 2019 international clinical trial of glecaprevir and pibrentasvir (G/P) treatment in adolescents with chronic hepatitis C virus (HCV) infection, demonstrated high efficacy and safety. However, few reports have considered real-world experience with G/P treatment in adolescents with chronic HCV. The present prospective multicenter study assessed real-world efficacy and safety of G/P treatment in Japanese adolescents with chronic HCV.
Subjects between 12 and 17 years old who were treatment-naïve or previously managed with interferon-based regimens were prospectively enrolled and treated with G/P (300 mg/120 mg) once daily for 8 or 12 weeks. The primary efficacy endpoint was sustained virologic response at 12 weeks after treatment completion (SVR12). Adverse effects and laboratory abnormalities were assessed.
Twenty-five Japanese patients (15 female) were enrolled from 13 pediatric centers in Japan. Median age was 13 years (range 12-17). Numbers of patients with genotypes 1b, 2a, 2b, and 2b/1b were 6, 12, 6, and 1, respectively. Twenty-two were treatment-naïve, while three had experienced interferon-based treatments. All patients completed G/P treatment (24 for 8 weeks and 1 for 12). Twenty-four achieved SVR12 (96%). Most adverse events were mild. None were serious. G/P significantly decreased serum alanine aminotransferase, γ-glutamyltransferase, and Wisteria floribunda agglutinin-positive Mac-2-binding protein concentrations. No negative effects on growth or maturation were apparent at 12 weeks.
Under real-world conditions, G/P treatment of Japanese adolescents with chronic HCV was highly efficacious and well tolerated.
DORA研究的第一部分是一项2019年针对慢性丙型肝炎病毒（HCV）感染青少年进行的glecaprevir和pibrentasvir（G/P）治疗的国际临床试验，显示出高疗效和安全性。然而，很少有报告考虑过G/P治疗慢性HCV青少年的真实世界经验。本前瞻性多中心研究评估了G/P治疗日本慢性HCV青少年的真实世界疗效和安全性。
前瞻性纳入12至17岁初治或先前接受基于干扰素方案治疗的受试者，给予G/P（300mg/120mg）每日一次，治疗8或12周。主要疗效终点是治疗完成后12周的持续病毒学应答（SVR12）。评估不良反应和实验室异常情况。
从日本13个儿科中心招募了25名日本患者（15名女性）。中位年龄为13岁（范围12 - 17岁）。基因型1b、2a、2b和2b/1b的患者数量分别为6、12、6和1。22例为初治患者，3例曾接受基于干扰素的治疗。所有患者均完成G/P治疗（24例治疗8周，1例治疗12周）。24例实现SVR12（96%）。大多数不良事件为轻度。无严重不良事件。G/P显著降低血清丙氨酸氨基转移酶、γ-谷氨酰转移酶和紫藤凝集素阳性Mac-2结合蛋白浓度。12周时对生长或成熟无明显负面影响。
在真实世界条件下，G/P治疗日本慢性HCV青少年具有高效性和良好耐受性。
