Efficacy and safety of treatment with sofosbuvir/velpatasvir in patients aged 6–18 years with chronic hepatitis C—Results of the PANDAA‐PED study

Abstract

Background and AimsThe aim of this non‐commercial, open‐label, real‐life, non‐randomized clinical trial was to analyse the efficacy and safety of a pangenotypic regimen sofosbuvir/velpatasvir (SOF/VEL) in patients aged 6–18 years with chronic hepatitis C virus (HCV) infection.MethodsFifty patients qualified for the 12‐week treatment were divided into two weight groups: 15 children weighting between 17 and <30 kg received a fixed dose of 200/50 mg of SOF/VEL (tablet) once daily, and 35 patients weighting ≥30 kg were treated with 400/100 mg SOF/VEL. The primary endpoint of the study was efficacy defined as sustained viral response (undetectable HCV RNA using an real‐time polymerase chain reaction method) at 12 weeks posttreatment (SVR12).ResultsMedian age of the participants was 10 (IQR 8–12) years, 47 were infected vertically, and 3 patients were previously ineffectively treated with pegylated interferon and ribavirin. Thirty‐seven participants were infected with HCV genotype 1, 10 with HCV genotype 3 and the remaining 3 with genotype 4. There was no case of cirrhosis. SVR12 was 100%. Thirty‐three reported adverse events (AEs) were considered related to the administration of SOF/VEL, all of them were mild or moderate. Children presenting with AEs were older compared to these without AEs: 12 (9.5–13) versus 9 (IQR 8–11) years (p = 0.008).ConclusionsResults of the PANDAA‐PED study indicated a 100% effectiveness of a 12‐week therapy with SOF/VEL in children aged 6–18 years with chronic HCV infection and its good safety profile, in particular in younger patients.

Profile of Sofosbuvir and Velpatasvir Combination in the Treatment of Chronic Hepatitis C in Children and Adolescents: Current Evidence

“…Similar data were found in the study by Pokorska-Spiewak et al for children 6–11 years of age. 25 These findings support the consideration of the use of sofosbuvir and velpatasvir combination therapy as a viable treatment option for young children with chronic hepatitis C.…”

“… 28 A single center open-label study of 50 children and adolescents 6–18 years of age with HCV treated with SOF/VEL demonstrated 100% SVR at 12 weeks. 25 In this study, adverse events possibly or probably related to SOF/VEL were mild to moderate (headache, abdominal pain and asthenia were the most common) and were more common in the adolescents and none led to discontinuation of treatment. These observed virologic responses are comparable to the 95–99% SVR rates in the adult trials 22 , 29 and demonstrate the potency and effectiveness of this combination in eradicating the virus and preventing disease progression.…”

“… 20 , 23 , 24 Preliminary data in children and adolescents suggest that it has similar efficacy to that seen in adults. 25 Its unique mechanism of action, high efficacy rates across all HCV genotypes, and favorable safety profile make it a consideration for first-line therapy. Individualized dosing and treatment duration studies are still needed to ensure optimal outcomes in the pediatric population.…”

ESPGHAN recommendations on treatment of chronic hepatitis C virus infection in adolescents and children including those living in resource‐limited settings

“…The results of the registration study have been confirmed in a recent real-world study using the same drug doses and treatment durations in adolescents. 16…”