Efficacy and safety of sofosbuvir-based therapies for treating HCV in children: observations from a real-world...

The aim of this study was to analyze the safety and efficacy of sofosbuvir-based regimens (SOF/velpatasvir and SOF/ledipasvir, SOF/VEL and SOF/LDV) for the treatment of hepatitis C in children aged 5-17 years in a real-life setting.

All participants who received SOF-based regimens beginning in August 2019 were included. The treatment duration was 12 weeks for both the SOF/LDV and SOF/VEL regimens; however, in the case of children infected with HCV genotype 1 and presenting with cirrhosis, the duration of the SOF/LDV treatment was extended to 24 weeks. In all cases, fixed doses of SOF/LDV and SOF/VEL were used adjusted to the patients' age and weight. The primary endpoint for these studies included evaluation of treatment efficacy (defined as sustained virologic response, SVR12, with undetectable HCV RNA at 12 weeks posttreatment).

We identified 90 patients aged 5 to 17 years eligible for the study: 50 who were treated with SOF/VEL and 40 who received SOF/LDV. The SVR12 assessment was available for 89 patients. In all of them, SVR12 was achieved (100% efficacy in per-protocol analysis; 99% efficacy in intention-to-treat assessment). One-third of the study participants reported adverse events (AEs) related to antiviral treatment, all of which were mild or moderate. Headache, fatigue, and abdominal pain were the most common symptoms. Some differences in the frequency of AEs according to the DAA used were noted (abdominal pain and asthenia were less common in children treated with SOF/LDV than in those treated with SOF/VEL). No case of treatment discontinuation occurred.

This study confirms the excellent efficacy and good tolerability of SOF-based regimens for the treatment of hepatitis C in children aged 5 to 17 years in real-life settings, supporting the inclusion of pediatric patients in national treatment policies.