Chronic hepatitis C virus (HCV) infection has been linked to faster liver disease development and a greater risk of malignancy relapse owing to chemotherapy discontinuation in children with hematological malignancies. For 12 weeks, researchers assessed the safety and effectiveness of ledipasvir-sofosbuvir in these individuals. Patients with chronic HCV genotype 1 or 4 infections undergoing maintenance chemotherapy for hematological malignancies got ledipasvir-sofosbuvir (90 mg/400 mg) once daily for 12 weeks in phase 2, an open-label trial at one location. The efficacy objective was the sustained virologic response 12 weeks following therapy (SVR12). The incidence of adverse events and clinical and laboratory data, including HCV flares defined as alanine aminotransferase >3-fold rise from Day 1 and HCV RNA elevation >1 log10 from Day 1, were used to assess safety. 

The median age of the 19 adolescents recruited and treated was 14 years (range 12–17), 84% (16/19) were male, and all had HCV genotype 4 and were HCV treatment naïve. All patients finished therapy and attained SVR12 (19/19, 100%, 95% CI, 82–100). The most common adverse effects were pyrexia (5/19, 26%), diarrhea (4/19, 21%), and headache (4/19, 21%). Three individuals had major adverse effects, including pneumonia (two patients) and osteoarthritis and diarrhea (one patient); none were thought to be connected to the study medicine. There were no HCV flares in any of the patients.

Ledipasvir-sofosbuvir was well-tolerated and effective in adolescents taking maintenance chemotherapy for chronic HCV genotype 4 with leukemia. The findings lent support to using an interferon-free regimen in adolescents with hematological malignancies.