Adolescents With HCV Achieve 98% Cure Rate in Direct-Acting Antiviral Study

Adolescents treated for hepatitis C achieved a 98% cure rate with a direct-acting antiviral drug (DAA) therapy, a study has found.

An all-oral combination of sofosbuvir (Sovaldi) and ribavirin was given to 52 people, aged 12 to 17 years old, with HCV genotype 2 or 3.

All of the 13 patients with genotype 2 achieved sustained virologic response (SVR) after 12 weeks of treatment, according to the findings. For those with genotype 3, the SVR rate was 97%, with 1 patient lost during follow-up.

“We found that children and adolescents respond quite well to these drugs with elimination of HCV and these drugs are quite safe,” a study author, Philip Rosenthal, MD, director of pediatric clinical research at the University of California, San Francisco, told MD Magazine.

DAA drugs have only recently been authorized for use by young patients.

The standard of care for chronic HCV in the pediatric population has been treatment for 24 or 48 weeks with interferon or peginterferon and ribavirin. This regimen requires injections and is associated with significant side effects such as growth impairment, according to the authors.

The US Food and Drug Administration on April 7 approved supplemental applications for sofosbuvir and ledipasvir and sofosbuvir (Harvoni) to treat HCV in children ages 12 to 17. Harvoni and Sovaldi were previously approved for adults.

These are the first direct-acting antiviral treatments approved for children and adolescents with HCV. DAA drugs reduce the amount of HCV in the body by preventing the virus from multiplying. In most cases, they cure HCV, according to the FDA.

The US Centers for Disease Control and Prevention (CDC) estimates 2.7 million to 3.9 million people in the US have chronic HCV. Children born to HCV-positive mothers are at risk for the liver-attacking virus. Some 23,000 to 46,000 children in the US are thought to have HCV infection.

“These approvals will help change the landscape for HCV treatment by addressing an unmet need in children and adolescents,” Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, said at the time of the drugs’ approval.

The study took place at 30 sites in Australia, Germany, Italy, New Zealand, Russia, the UK and the US from October 2014 to June 2016. The median age of the patients was 15 years. Seventy-five percent had genotype 3 and 83% had never been treated. Forty percent were assessed as not having cirrhosis; the remainder did not have a cirrhosis determination.

The most commonly reported adverse events were nausea (27%) and headache (23%), according to the authors.

Limitations of the study, which was supported by Gilead, include a small sample size and an incomplete cirrhosis assessment of the patients.

“It is unclear whether cirrhosis affects the response to sofosbuvir and ribavirin in adolescents,” the researchers wrote.

Rosenthal said he’s pleased that the FDA approved the DAA treatment for young people — and that it works.

“We were not surprised to find how efficacious and safe these drugs are,’’ Rosenthal said. “These children and adolescents deserve therapies just like adults.”

The study, "Sofosbuvir and ribavirin in adolescents 12-17 years old with hepatitis C virus genotype 2 or 3 infection," was published online in Hepatology last month.