Efficacy and Safety of Glecaprevir/Pibrentasvir in Children Aged 3-11 Years With Chronic Hepatitis C: A Real-World, Prospective, Multicenter Study in Japan.

Aim: 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.

Methods: 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.

Results: 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.

Conclusions: In real-world clinical practice, G/P treatment demonstrated high efficacy and good tolerability in children aged 3-11 years with chronic HCV.