Sofosbuvir–velpatasvir in children 3–17 years old with hepatitis C virus infection
(2024) Journal of Pediatric Gastroenterology and Nutrition — Vol. 78, n° 6, p. 1342-1354 (2024)
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Sokal, Etienne 
- Abstract
- (en) BACKGROUND: The safety and efficacy of sofosbuvir-velpatasvir in children aged 3-17 years with chronic hepatitis C virus (HCV) infection of any genotype were evaluated. METHODS: In this Phase 2, multicenter, open-label study, patients received once daily for 12 weeks either sofosbuvir-velpatasvir 400/100 mg tablet (12-17 years), 200/50 mg low dose tablet or oral granules (3-11 years and ≥17 kg), or 150/37.5 mg oral granules (3-5 years and <17 kg). The efficacy endpoint was sustained virologic response 12 weeks after therapy (SVR12). Dose appropriateness was confirmed by intensive pharmacokinetics in each age group. FINDINGS: Among 216 patients treated, 76% had HCV genotype 1% and 12% had genotype 3. Rates of SVR12 were 83% (34/41) among 3-5-year-olds, 93% (68/73) among 6-11-year-olds, and 95% (97/102) among 12-17-year-olds. Only two patients experienced virologic failure. The most common adverse events were headache, fatigue, and nausea in 12-17-year-olds; vomiting, cough, and headache in 6-11-year-olds; and vomiting in 3-5-year-olds. Three patients discontinued treatment because of adverse events. Four patients had serious adverse events; all except auditory hallucination (n = 1) were considered unrelated to study drug. Exposures of sofosbuvir, its metabolite GS-331007, and velpatasvir were comparable to those in adults in prior Phase 2/3 studies. Population pharmacokinetic simulations supported weight-based dosing for children in this age range. INTERPRETATION: The pangenotypic regimen of sofosbuvir-velpatasvir is highly effective and safe in treating children 3-17 years with chronic HCV infection.
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Citations
Jonas, M. M., Romero, R., Rosenthal, P., Lin, C.-H., Verucchi, G., Wen, J., Balistreri, W. F., Whitworth, S., Bansal, S., Leung, D. H., Narkewicz, M. R., Gonzalez‐Peralta, R. P., Mangia, A., Karnsakul, W., Rao, G. S., Shao, J., de Jong, J., Parhy, B., Osinusi, A., et al. (2024). Sofosbuvir–velpatasvir in children 3–17 years old with hepatitis C virus infection. Journal of Pediatric Gastroenterology and Nutrition, 78(6), 1342-1354. https://doi.org/10.1002/jpn3.12045 (Original work published 2024)