Ledipasvir/sofosbuvir combination resulted in beneficial viral response in pediatric patients.
An investigational dose of ledipasvir 45-mg/sofosbuvir 200-mg was found to cure nearly all children with hepatitis C virus (HCV) in a clinical trial, according to a study presented at the International Liver Congress.
The researchers discovered that 99% of patients aged 6 to 11 treated with the experimental combination had undetectable levels of HCV-RNA only 12 weeks after treatment. The combination was also found to be well-tolerated, with no patients experiencing serious adverse events.
Approximately 180 million individuals have HCV around the world, with children accounting 0.05% to 0.36% of that population in the United States and Europe. In developing countries, pediatric patients can account for 1.8% to 5.8% of HCV infections.
Direct-acting antivirals (DAA) are the standard treatment for adults with HCV, but pediatric patients have long been treated for 24 to 48 weeks with pegylated interferon plus ribavirin (RBV). This treatment can result in severe adverse events.
"Direct-acting antivirals have transformed the treatment of adults with chronic HCV, however, studies of these new therapies in children are required," said lead study author Karen Murray, MD. "These data establish the use of the oral direct-acting antivirals as an important treatment option in HCV-infected children aged six to 11 years old."
The ongoing clinical trial included 90 pediatric patients aged between 6 and 11 with HCV. Of these patients, 86 had HCV genotype 1. These patients receive 12 weeks of treatment. Patients with cirrhosis or had failed previous treatment with pegylated interferon plus RBV received 24 weeks of treatment, according to the study.
Patients with HCV genotype 3 received treatment with ledipasvir/sofosbuvir plus RBV for 24 weeks, while patients with genotype 4 received treatment with ledipasvir/sofosbuvir for 12 weeks.
The authors reported that a majority of patients were male, white, and treatment-naïve. Nearly all patients developed the infection as a result of mother-to-child transmission, according to the study.
The investigators found that 89 of the 90 patients sustained a virologic response at 12 weeks. Only 1 HCV genotype 1 patient with cirrhosis relapsed. These findings show that this experimental combination may be a promising treatment for pediatric patients with HCV, according to the authors.
The authors reported that no patients discontinued the treatment due to drug-related severe or life-threatening adverse events, according to the study. Common side effects included headache, fever, abdominal pain, diarrhea, vomiting, cough, fatigue, sore throat, and nausea.
These positive results may present a new treatment option to pediatric patients that is more effective and comes with less side effects than current treatments.
"This study is a breakthrough for the management of children aged six to 11 years old with hepatitis C, demonstrating that the new DAA regimen is highly efficacious and, more importantly, safe in this group of HCV-infected children," said Frank Tacke, University Hospital Aachen, Germany, and European Association for the Study of the Liver Governing Board Member.