The pangenotypic direct-acting antiviral Mavyret demonstrated 100% sustained virologic response in adolescent patients aged 12 years to 17 years with a safety profile consistent with adult patients, according results from part one of the DORA study.
Researchers designed the DORA study to evaluate the pharmacokinetics, safety, and efficacy of Mavyret (glecaprevir/pibrentasvir, AbbVie) in pediatric patients with chronic HCV infection. The second part of the study will evaluate pediatric formulation for patients aged 3 years to 11 years.
“Although the symptoms of chronic HCV infection in the pediatric population are usually mild, the consequences of disease progression in children and adolescents are similar to those in adults,” Maureen M. Jonas, MD, from the Center for Childhood Liver Disease at the Boston Children’s Hospital, Massachusetts, and colleagues wrote. “The goals and endpoint of therapy are therefore the same regardless of age — cure of infection, and prevention of progression of HCV infection, such as HCV-related liver disease.”
The first part of the study comprised 48 patients, 77% of whom were treatment naive. Patients with treatment experience previously received pegylated interferon with ribavirin. Most patients received 8 weeks of therapy (94%), while three patients received 16 weeks of therapy.
No patients discontinued treatment and there were no virologic failures. Additionally, the presence of NS3 and NS5A resistance had no impact on SVR.
Most adverse events were mild and unrelated to treatment. The researchers noted that treatment did not appear to have an impact on growth and development.
Of the 44 patients with available data on patient-reported quality of life PedsQL score, mean change from baseline in psychosocial health summary score improved by 2.4 points and the mean change from baseline in physical health summary score improved by 2 points for an overall improvement of 2.3 points.
“Treatment and management of chronic HCV infection in adolescent population will not only mitigate the development of progressive liver injury, but also prevent further transmission of HCV,” Jonas and colleagues wrote. “To achieve this goal, a short duration pangenotypic IFN- and RBV-free treatment option, with improved tolerability and high SVR rates, could provide assurance that early treatment is a viable option, rather than deferring treatment until adulthood.” – by Talitha Bennett
Disclosure: Jonas reports she is a consultant for and received grant support from Gilead; and received grant support from AbbVie and Bristol-Myers Squibb. Please see the full study for all other authors’ relevant financial disclosures.