Investigational Ledipasvir/Sofosbuvir Dose Safe in Children With HCV
The SVR4 in the 88 patients who completed the post-treatment Week 4 visit was 99%.
An investigational dosage of once-daily ledipasvir/sofosbuvir (LDV/SOF) plus ribavirin (RBV) for 12 or 24 weeks was safe and highly efficacious in children age 6 to 11 with chronic hepatitis C virus (HCV) infection, according to research presented at the 2017 International Liver Congress™ held April 19-23 in Amsterdam, The Netherlands.
Karen F. Murray, MD, division chief of pediatric gastroenterology and hepatology at the Seattle Children's Hospital in Washington, and colleagues reviewed data from 90 children between age 6 and 11 enrolled in an open-label ongoing study. Patients received LDV/SOF in a 45 mg/200 mg fixed-dose combination tablet plus RBV (15 mg/kg/day up to 1400 mg/day) for 12 or 24 weeks. The appropriateness of the LDV/SOF dose was confirmed with a pharmacokinetic sampling on day 10 in the first 12 patients.
Of the 90 children, the majority (n=86) had HCV genotype 1 infection with the remaining 4 having genotype 3 (n=2) and genotype 4 (n=2) infection. Mean age was 9, mean weight was 33 kg (range 18 to 76 kg), and mean body mass index (BMI) was 18 kg/m2 (range 13 to 31 kg/m2). Most of the children were male (59%), white (79%), treatment naïve (80%), and vertically infected (97%).
Of the 88 patients who completed the 4-week post-treatment follow-up visit, the sustained virologic response rate at 4 weeks (SVR4) was 99% (87/88); 1 patient relapsed.
The most common side effects reported in 10% or more of patients were abdominal pain, headache, diarrhea, vomiting, nausea, fatigue, pyrexia, cough, and oropharyngeal pain. No patient experienced a Grade 3-4 adverse event (AE) or AEs leading to study drug discontinuation.
"This all-oral, interferon-free regimen was well tolerated, supporting its potential as an important treatment option for children 6 to 11 years old," concluded the researchers.
The study is continuing in children age 3 to 5.
The researchers report financial relationships with AbbVie, Albireo, Alexion Pharmaceuticals Inc, Bristol-Myers Squibb, Genentech, Inc, Gilead Sciences, Merck & Co, Inc, Retrophin, Inc, Roche, Sucampo Pharmaceuticals, Inc, Shire, and Vertex Pharmaceuticals.
Murray KF, Balistreri W, Bansal S, et al. Ledipasvir/sofosbuvir ± ribavirin for 12 or 24 weeks is safe and effective in children 6-11 years old with chronic hepatitis C infection. Presented at: The International Liver Congress™ 2017. Amsterdam, The Netherlands; April 19-23, 2017. Abstract PS-101.