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Published on: March 22, 2011
Sofosbuvir-velpatasvir in children 3-17 years old with hepatitis C virus infection
Maureen M Jonas1, Rene Romero2, Philip Rosenthal3
1Boston Children's Hospital, Boston, Massachusetts, USA.
Insights
Sofosbuvir-velpatasvir effectively treats chronic hepatitis C virus (HCV) in children aged 3-17. This pangenotypic regimen demonstrated high sustained virologic response rates and a favorable safety profile in pediatric patients.
Area of Science:
- Hepatology
- Pediatric Infectious Diseases
- Virology
Background:
- Chronic hepatitis C virus (HCV) infection affects children globally.
- Limited treatment options exist for pediatric HCV patients.
Purpose of the Study:
- To evaluate the safety and efficacy of sofosbuvir-velpatasvir in children aged 3-17 with chronic HCV.
- To determine appropriate weight-based dosing for pediatric patients.
Main Methods:
- Phase 2, multicenter, open-label study.
- 12-week treatment with sofosbuvir-velpatasvir using age and weight-based dosing.
- Sustained virologic response 12 weeks after therapy (SVR12) as the primary efficacy endpoint.
- Pharmacokinetic analysis to confirm dose appropriateness.
Main Results:
- High SVR12 rates observed across age groups: 83% (3-5 yrs), 93% (6-11 yrs), and 95% (12-17 yrs).
- Virologic failure occurred in only two patients.
- Generally well-tolerated, with most common adverse events including headache, fatigue, and vomiting.
- Pharmacokinetic exposures were comparable to adults, supporting weight-based dosing.
Conclusions:
- Sofosbuvir-velpatasvir is a highly effective and safe pangenotypic treatment for chronic HCV in children aged 3-17.
- The study supports the use of weight-based dosing for pediatric HCV treatment.
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.
