Efficacy and Safety of Sofosbuvir-Based Therapies in Pediatric Hepatitis C
A RECENT study has demonstrated the high efficacy and favorable safety profile of sofosbuvir-based regimens in treating hepatitis C virus (HCV) in children aged 5 to 17 years. The research, conducted in a real-world setting, offers strong evidence for the inclusion of these treatments in pediatric care protocols.
Study Design and Methodology
The study enrolled 90 children, all of whom received a 12-week course of either sofosbuvir/velpatasvir (SOF/VEL) or sofosbuvir/ledipasvir (SOF/LDV) regimens. The treatment duration was extended to 24 weeks for patients with HCV genotype 1 and cirrhosis. All doses were adjusted based on the patient’s age and weight, ensuring the most appropriate treatment for each individual. The primary endpoint of the study was achieving sustained virologic response at 12 weeks (SVR12), defined as undetectable HCV RNA levels.
Key Findings: 100% Efficacy in Pediatric HCV Treatment
Results showed that all 89 patients assessed for SVR12 reached the target outcome, resulting in a 100% success rate in the per-protocol analysis. In the intention-to-treat analysis, the efficacy remained high at 99%. The findings support the efficacy of sofosbuvir-based therapies as first-line treatments for pediatric HCV, with no patients discontinuing treatment due to adverse events (AEs).
Adverse Events and Safety Profile
While approximately one-third of participants reported adverse events, these were generally mild or moderate. The most common side effects included headache, fatigue, and abdominal pain, with differences in symptom frequency noted between the two treatment regimens. Children treated with SOF/LDV experienced fewer cases of abdominal pain and asthenia compared to those treated with SOF/VEL.
Conclusion: A Milestone for Pediatric Hepatitis C Treatment
This study adds to the growing body of evidence that supports the use of sofosbuvir-based regimens in treating pediatric patients with HCV. The 100% efficacy rate and the favorable safety profile underscore the potential for these treatments to be included in national pediatric HCV treatment guidelines. By providing robust treatment options for young patients, healthcare providers can significantly improve outcomes for this vulnerable group.
Reference: Pokorska-Śpiewak M et al. Efficacy and safety of sofosbuvir-based therapies for treating HCV in children: observations from a real-world setting. Eur J Clin Microbiol Infect Dis. 2025; doi: 10.1007/s10096-025-05320-4.
