Just five months after a clinical trial demonstrated its effectiveness in children and adolescents, a combination drug therapy for the hepatitis C virus (HCV) infection was approved by the Food and Drug Administration for patients 12 and older with the disease. Cincinnati Children’s pediatric gastroenterologist William Balistreri, MD, was the lead author of the article reporting the results of the trial, which assessed the efficacy of ledipasvir-sofosbuvir in children infected with HCV. The ledipasvir-sofosbuvir combination is the first all-oral, direct-acting antiviral regimen approved for children with chronic HCV infection, and represents a significant improvement in care for patients who previously had limited treatment options.

The phase two, multicenter, open-label study was conducted between November 2014 and October 2015 at 24 sites across the United States, the United Kingdom and Australia. One hundred 12- to 17-year-olds received ledipasvir-sofosbuvir once daily for 12 weeks in a dosage that had already proved effective in adults. Two patients did not complete the treatment and were lost to follow-up, but all 98 patients who completed the trial successfully reached a virologic response. In fact, following treatment, blood tests showed no trace of the virus in those study participants.

In April 2017, the FDA approved supplemental indications for the therapy for adolescents with any of the six genotypes of chronic HCV infection. When prescribed to children with genotype 1, 4, 5 or 6, the drug is called Harvoni (a combination of ledipasvir-sofosbuvir). When prescribed for children with genotype 2 or 3, it is called Sovaldi (sofosbuvir only) and given in combination with ribavirin.

“There are an estimated 23,000 to 46,000 pediatric HCV patients in the United States, most of whom were infected with the virus at birth,” says Balistreri. 

Cases of HCV infection have been increasing for several years among those who inject opioids, and infections have reached epidemic proportions in most states among this population. “In the Cincinnati area alone, there has been an increase of more than 350 percent in HCV infection among people 12 to 29 years old, which of course includes women of childbearing age who may pass the infection on to their newborn infants,” Balistreri says. “We estimate that there has been a 450 percent rise in cases of HCV among children and adolescents at Cincinnati Children’s since 2009. With this in mind, standard screening for HCV in pregnant women may be appropriate.”

The study, “The Safety and Effectiveness of Ledipasvir−Sofosbuvir in Adolescents 12-17 Years Old with Hepatitis C Virus Genotype 1 Infection,” was published online in Hepatology ahead of the journal’s August 2017 issue.

Read the abstract