Pokorska-Śpiewak Maria, Dobrzeniecka Anna, Aniszewska Małgorzata, Marczyńska Magdalena
Department of Children's Infectious Diseases, Regional Hospital of Infectious Diseases in Warsaw, Medical University of Warsaw, Wolska Str. 37, 01-201 Warsaw, Poland.
Department of Pediatric Infectious Diseases, Regional Hospital of Infectious Diseases in Warsaw, 01-201 Warsaw, Poland.
J Clin Med. 2021 Sep 15;10(18):4176. doi: 10.3390/jcm10184176.
Available real-world data on the efficacy and safety of ledipasvir/sofosbuvir (LDV/SOF) in pediatric patients are limited. In this prospective, open-label, single-center study, we aimed to present our real-life experience with a fixed dose of LDV/SOF (90/400 mg) for the treatment of chronic hepatitis C (CHC) genotypes 1 and 4 in children aged 12 to 17 years.
We analyzed intention-to-treat (ITT) and per-protocol (PP) rates of sustained virological response (SVR), defined as undetectable HCV viral load at posttreatment week 12, in 37 participants treated with LDV/SOF according to the HCV genotype, baseline liver fibrosis, duration of treatment, and experience of the previous ineffective antiviral treatment. There were 32 patients infected with genotype 1 and 5 with genotype 4. Fourteen (38%) participants were treatment-experienced, two were coinfected with HIV, and three were cirrhotic. Two patients qualified for 24 weeks of therapy, and the remaining 35 received 12 weeks of LDV/SOF treatment.
The overall ITT SVR12 rate was 36/37 (97%). One patient was lost to follow-up after week 4 of therapy when his HCV RNA was undetectable. All 36 patients who completed the full protocol achieved SVR (36/36, 100%). PP analyses of SVR12 rates according to the HCV genotype, baseline liver fibrosis, duration of the treatment, and previous ineffective treatment were all 100%. A significant decrease in aminotransferase serum levels was observed in the subsequent weeks of the treatment and at SVR assessment compared to baseline. No serious adverse events were reported.
The results of this study confirm previous observations of a suitable efficacy and safety profile of LDV/SOF for the treatment of CHC genotypes 1 and 4 in adolescents.
关于来迪派韦/索磷布韦（LDV/SOF）在儿科患者中的疗效和安全性的真实世界数据有限。在这项前瞻性、开放标签、单中心研究中，我们旨在介绍我们使用固定剂量的LDV/SOF（90/400毫克）治疗12至17岁儿童慢性丙型肝炎（CHC）1型和4型的实际经验。
我们分析了37名接受LDV/SOF治疗的参与者的意向性治疗（ITT）和符合方案（PP）的持续病毒学应答（SVR）率，SVR定义为治疗后第12周丙型肝炎病毒（HCV）载量不可检测，根据HCV基因型、基线肝纤维化、治疗持续时间和先前无效抗病毒治疗的经验进行分析。有32例患者感染1型，5例感染4型。14名（38%）参与者有治疗史，2例合并感染HIV，3例为肝硬化患者。2例患者符合24周治疗标准，其余35例接受12周的LDV/SOF治疗。
总体ITT SVR12率为36/37（97%）。1例患者在治疗第4周后失访，当时其HCV RNA不可检测。所有36例完成完整方案的患者均实现了SVR（36/36，100%）。根据HCV基因型、基线肝纤维化、治疗持续时间和先前无效治疗进行的SVR12率的PP分析均为100%。与基线相比，在治疗的后续几周和SVR评估时观察到血清转氨酶水平显著下降。未报告严重不良事件。
本研究结果证实了先前关于LDV/SOF治疗青少年CHC 1型和4型具有适宜疗效和安全性的观察结果。
