SOF/VEL combo improves well-being of children with CHC

In children with chronic hepatitis C (CHC), effective treatment with sofosbuvir/velpatasvir (SOF/VEL) results in improvements in certain areas of their well-being at 1 year. This suggests some patient-reported outcomes (PRO) benefits of direct-acting antiviral (DAA) therapy. However, the parents’ proxy reports show a decrease in the patients’ school functioning.

“Despite the improvement in the child self-report of ‘autonomy & parent relation’, there was a more pronounced discrepancy between children self-reports and parents proxy reports in all dimensions of health-related quality of life (HRQOL),” the researchers said. “Older patients’ age correlated with worse HRQOL assessment.”

In the nonrandomized, open-label PANDAA-PED study, 50 patients who received treatment with a fixed dose of SOF/VEL achieved sustained virologic response at 12 weeks after the end of treatment.

The researchers compared the HRQOL at 1 year after the treatment with the baseline assessment (before the treatment). They used the KIDSCREEN-27 questionnaires, which included five dimensions of HRQOL, for child self-reporting and parent proxy reporting. The normal range was set to T values of 40–60 points. The intraclass correlation coefficient (ICC) was used to analyse child-parent agreement.

Mean T values for all HRQOL dimensions were within the normal range, with a significant improvement seen in “autonomy & parent relation,” from 48.3 to 51.1 (p=0.03), in the self-assessment report of children. [Pediatr Infec Dis J 2025;44:405-410]

On the other hand, parent proxy assessment revealed a significant reduction in the “school” dimension, from 49.5 to 45.8 (p=0.03), which did not appear at 3-month post-treatment. In addition, older age showed a significant association with worse HRQOL scores in all dimensions.

In the assessment of ICC for child self-reports and parent proxy reports, agreement was poor to moderate for most single measures and lower than that at 3-month post-treatment analysis.

Recommended DAA

The SOF/VEL combination therapy has been shown to be safe and effective in children as young as 3 years old, making it a preferable pangenotypic DAA recommended for CHC treatment in paediatric patients. [J Pediatr Gastroenterol Nutr 2024;78:957-972; Liver Int 2024;44:663-681; Liver Int 2023;43:1871-1878; J Pediatr Gastroenterol Nutr 2024;78:1342-1354]

However, in a study showcasing the treatment effects of SOF/VEL in 216 children with hepatitis C virus (HCV) infection, two had serious psychiatric adverse events of suicidal ideation with one suicidal attempt and bipolar disorder. Both cases occurred in adolescents with complex medical history and could be unrelated to the study drug. [J Pediatr Gastroenterol Nutr 2024;78:1342-1354]

“Thus, it seems essential to analyse the influence of DAA treatment not only on liver disease but also on PROs, including HRQOL,” the researchers said. 

"We documented a significant increase in self-reported physical well-being and a trend toward higher social support and peers scores in parent evaluations, [as well as] an increasing proportion of children who estimated that their physical well-being improved,” they added.

“These results supported the hypothesis that successful treatment of HCV led not only to clinical improvement but also to benefits for PROs also in children and adolescents,” the researchers said.