Long-term efficacy and safety of sofosbuvir-based direct-acting antiviral regimens in paediatric patients with hepatitis C virus infection: an international registry study.

Background: Direct-acting antiviral treatment is associated with high rates of sustained virological response and has a favourable safety profile in children aged 3-17 years with chronic hepatitis C virus (HCV) infection, but data describing the durability of sustained virological response and its effects on growth and sexual development are needed. This study aimed to assess the efficacy and long-term effects of sofosbuvir-based direct-acting antiviral regimens on growth and development in the paediatric population.

Methods: In this international, multicentre, observational registry study, children aged 3-17 years with HCV who received sofosbuvir plus ribavirin, ledipasvir plus sofosbuvir with or without ribavirin, sofosbuvir plus velpatasvir, or sofosbuvir plus velpatasvir plus voxilaprevir in previous clinical trials were eligible for follow-up of growth and sexual development parameters for up to 5 years.

Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial.

Background: In patients with Alagille syndrome, cholestasis-associated clinical features can include high serum bile acids and severe pruritus that can necessitate liver transplantation. We aimed to evaluate the efficacy and safety of the ileal bile acid transporter inhibitor odevixibat versus placebo in patients with Alagille syndrome.

Methods: The ASSERT study was a phase 3, double-blind, randomised, placebo-controlled trial that enrolled patients at 21 medical centres or hospitals in ten countries (Belgium, France, Germany, Italy, Malaysia, the Netherlands, Poland, Türkiye, the UK, and the USA).

Prospective study of quantitative liver MRI in cystic fibrosis: feasibility and comparison to PUSH cohort ultrasound.

Background: Pediatric radiologists can identify a liver ultrasound (US) pattern predictive of progression to advanced liver disease. However, reliably discriminating these US patterns remains difficult. Quantitative magnetic resonance imaging (MRI) may provide an objective measure of liver disease in cystic fibrosis (CF).

Long-term follow-up and liver outcomes in children with cystic fibrosis and nodular liver on ultrasound in a multi-center study.

Background: Nodular liver (NOD) in cystic fibrosis (CF) suggests advanced CF liver disease (aCFLD); little is known about progression of liver disease (LD) after detection of sonographic NOD.

Methods: Clinical, laboratory, and ultrasound (US) data from Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in CFLD Study participants with NOD at screening or follow-up were compared with normal (NL). Linear mixed effects models were used for risk factors for LD progression and Kaplan-Meier estimator for time-to-event.

Association Between Transient Elastography and Controlled Attenuated Parameter and Liver Ultrasound in Children With Cystic Fibrosis.

Methods to identify children with cystic fibrosis (CF) at risk for development of advanced liver disease are lacking. We aim to determine the association between liver stiffness measurement (LSM) by vibration-controlled transient elastography (VCTE) with research ultrasound (US) patterns and conventional hepatic markers as a potential means to follow liver disease progression in children with CF. ELASTIC (Longitudinal Assessment of Transient Elastography in CF) is a nested cohort of 141 patients, ages 7-21, enrolled in the Prediction by US of Risk of Hepatic Cirrhosis in CF (PUSH) Study.

Impact of Genotype, Serum Bile Acids, and Surgical Biliary Diversion on Native Liver Survival in FIC1 Deficiency.

Background And Aims: Mutations in ATPase phospholipid transporting 8B1 (ATP8B1) can lead to familial intrahepatic cholestasis type 1 (FIC1) deficiency, or progressive familial intrahepatic cholestasis type 1. The rarity of FIC1 deficiency has largely prevented a detailed analysis of its natural history, effects of predicted protein truncating mutations (PPTMs), and possible associations of serum bile acid (sBA) concentrations and surgical biliary diversion (SBD) with long-term outcome. We aimed to provide insights by using the largest genetically defined cohort of patients with FIC1 deficiency to date.

The management of hepatitis B and hepatitis C in children.

In the present paper, we review the increased disease burden of hepatitis B (HBV) and hepatitis C (HCV) infection that is recognized worldwide; especially in children when the most common mode of transmission is vertically from infected mothers. In children with HBV and HCV infection, spontaneous clearance of the virus in the first years of life is not common, in contrast with adults, but these patients often stay asymptomatic until early adulthood, when disease has progressed to chronic hepatitis with increased risk of cirrhosis and its complication, and hepatocellular carcinoma. Due to limited treatment options of HBV infection in the pediatric population, clinicians focus on primary prevention, by vaccinating all infants during their first days of life.