I’ve treated children with hepatitis C, and watching them endure weekly interferon injections for up to two years was heartbreaking. So when the data from this new study was presented at the 2018 AASLD Liver Meeting in San Francisco, I felt a surge of hope for our youngest patients.
The development of these sofosbuvir/ledipasvir oral granules for children ages 3 to 6 represents a remarkable shift in pediatric HCV treatment. The formulation is brilliantly simple – parents can sprinkle it on ice cream or pudding (though we’re careful to specify non-acidic foods), and the dosing is precisely calculated based on weight. For children 17kg or above, we use 200mg sofosbuvir plus 45mg ledipasvir, while smaller children receive 150mg sofosbuvir plus 33.75mg ledipasvir, achieving drug exposure levels comparable to adult treatments.
Dr. Kathleen Schwarz’s team at Johns Hopkins conducted a fascinating study with 34 children across the UK, US, and Australia. What struck me most was the demographic pattern – 70% girls, 80% white, and every single case traced back to mother-to-child transmission. I think this reflects a growing concern in the US, where we’re seeing more HCV cases in children as the opioid epidemic affects more young women of childbearing age.
The results were nothing short of remarkable – 97% cure rate after just 12 weeks of treatment plus 12 weeks of follow-up. Only one child discontinued treatment, and that was due to the taste of the medication (which, honestly, is a common challenge when treating young children). Even the patients with resistance-associated mutations at baseline achieved cure, which I found particularly encouraging.
The side effect profile was surprisingly mild – mostly just typical childhood ailments like coughs, fevers, and runny noses. In my experience, this is worlds apart from the brutal side effects we see with interferon-based treatments. One of my young patients on the old regimen used to cry before each weekly injection, but these granules seem to be causing minimal disruption to children’s lives.
We’ve come such a long way since I first started treating pediatric HCV. The earlier approval of sofosbuvir and Harvoni (400/90mg tablets) for adolescents weighing at least 35kg was groundbreaking, and now we’re extending that same highly effective treatment to even younger children. In some regions, like Egypt, where pediatric HCV is far more common, this development could transform thousands of young lives.
If you’re considering this treatment for young patients, remember to calculate the dose carefully based on weight, and counsel parents about proper administration with food. Make sure to explain that while mixing with ice cream might seem indulgent, it’s actually crucial for proper absorption. And don’t forget to warn them about avoiding acidic foods for mixing – we’ve learned that lesson the hard way.