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Published on: June 20, 2020
Ledipasvir-Sofosbuvir for 12 Weeks in Children 3 to <6 Years Old With Chronic Hepatitis C
Kathleen B Schwarz1, Philip Rosenthal2, Karen F Murray3
1Johns Hopkins Hospital, Baltimore, MD.
Insights
Ledipasvir-sofosbuvir is a safe and effective treatment for young children with chronic hepatitis C virus (HCV) infection, achieving a 97% sustained virological response. This direct-acting antiviral offers a new option for pediatric HCV treatment.
Area of Science:
- Pediatric Gastroenterology
- Hepatology
- Infectious Diseases
- Virology
Background:
- No direct-acting antiviral (DAA) treatments are currently approved for children under 12 with chronic hepatitis C virus (HCV) infection.
- HCV infection in young children, often acquired perinatally, requires safe and effective therapeutic options.
Purpose of the Study:
- To evaluate the safety and efficacy of ledipasvir-sofosbuvir in children aged 3 to <6 years with chronic HCV infection.
- To assess the pharmacokinetic profile of ledipasvir-sofosbuvir in this pediatric population.
Main Methods:
- An open-label study involving 34 treatment-naïve children (3 to <6 years) with chronic HCV genotypes 1 or 4.
- Patients received weight-based doses of ledipasvir-sofosbuvir granules for 12 weeks.
- Sustained virological response 12 weeks post-treatment (SVR12) was the primary endpoint; pharmacokinetic sampling was conducted in a subset of patients.
Main Results:
- A high SVR12 rate of 97% (33 of 34 patients) was achieved.
- Ledipasvir-sofosbuvir was well tolerated, with the most common adverse events being vomiting, cough, and pyrexia; no serious adverse events were reported.
- Pharmacokinetic analysis confirmed the appropriateness of the selected weight-based doses.
Conclusions:
- Ledipasvir-sofosbuvir demonstrates high efficacy and favorable tolerability in young children (3 to <6 years) with chronic HCV infection.
- This DAA combination represents a promising treatment option for this underserved pediatric population.
- Further research may support expanded DAA approvals for younger pediatric age groups.
Abstract:
For children under 12 years of age who have chronic hepatitis C virus (HCV) infection, there are currently no approved treatments with direct-acting antiviral agents. We therefore evaluated the safety and efficacy of ledipasvir-sofosbuvir in HCV-infected children aged 3 to <6 years. In an open-label study, patients 3 to <6 years old chronically infected with HCV genotype 1 (n = 33) or 4 (n = 1) received weight-based doses of combined ledipasvir-sofosbuvir as granules (33.75 mg/150 mg for weights <17 kg or 45 mg/200 mg for weights ≥17 kg) for 12 weeks. The primary endpoint was sustained virological response 12 weeks after treatment (SVR12). For the first 14 patients, intensive pharmacokinetic sampling was done on day 10 of treatment. All patients had been infected through perinatal transmission and were treatment naïve. No patients had known cirrhosis. Ten patients (29%) weighed <17 kg. SVR12 was achieved in 97% of patients (33 of 34); the patient who did not achieve SVR12 was 3 years old and discontinued treatment after 5 days because of an adverse event "abnormal drug taste." The most common adverse events were vomiting (24% of patients), cough (21%), and pyrexia (21%). No patients experienced a serious adverse event. Intensive pharmacokinetic analysis of 13 patients for whom data were evaluable confirmed that the doses selected were appropriate. Conclusion: Ledipasvir-sofosbuvir was well tolerated and highly effective in children 3 to <6 years old with chronic HCV infection.
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