Recently, Dr. William Balistreri presented a review of some of the biggest advances in pediatric hepatology this past year on the Bowel Sounds Podcast (with the award-winning hosts).

He discussed the following:

• IBAT inhibitors which are a game-changer for pruritic cholestatic disorders like Alagille syndrome. By reducing itching, it may help many avoid liver transplantation
• HCV medications which usually result in a cure with typical therapy courses running 8-12 weeks
• Emergence of a new treatment, Fazirsiran, for alpha-one antitrypsin deficiency (see blog post below)
• More data showing the good liver safety of methotrexate in individuals without preexisting liver disease. Dr. Balistreri and colleagues showed pediatric patients with JRA did not develop liver fibrosis/clinical liver disease in 1997.
• How Gilbert’s may be beneficial –>hopeful news for the mildly jaundiced children that we see. Science (M Leslie, 6/8/23): Can ‘toxic’ bilirubin treat a variety of illnesses

He kindly agreed to send me a few slides on the later two subjects at my request:

Related blog posts:

IBAT Inhibitors:

• Six Year Data for IBAT Inhibitor Treatment for Alagille Syndrome
• Lecture: IBAT Inhibitor for Alagille Syndrome
• Aspen Webinar 2021 Part 3-IBAT Inhibitors
• GALA: Alagille Study

Hepatitis C

• Eradicating Hepatitis C Dependent on Congressional Action
• Hepatitis C is Undertreated in the U.S.
• Why CDC is Drafting New Guidelines for Screening Children for Perinatally-Acquired Hepatitis C Infection
• NASPGHAN Foundation: Hepatitis C in Children and Adolescents Lecture Slides
• Improvement in Liver Fibrosis with DAA Treatment of Hepatitis C in Adolescents
• Hepatitis C in 2020: NASPGHAN Position Paper
• Online Aspen Webinar (Part 4) -How to Treat Hepatitis C in Children
• Medical Progress: Toward Hepatitis C Elimination Detailed information on treatment of HCV
• Oral Pan-Genotypic HCV Drugs Approved For Children Starting at Age 3 Years

Alpha-One Antitrypsin

• RNA Interference (Fazirsiran) for Liver Disease Associated with Alpha-1-Antitrypsin Deficiency
• Alpha-1-Antitrypsin Deficiency (review May 2020). 35% of adults with ZZ genotype show clinically-significant liver fibrosis. Risk factors for advanced fibrosis: male gender, metabolic syndrome/obesity, and alcohol consumption.
• Liver Shorts March 2020 (A1AT Heterozygosity worsens NAFLD/contributes to cirrhosis)

Dr. Balistreri

• NASPGHAN22: History of Pediatric GI & Selected Slides from the William F Balistreri Lecture (Part 1)
• NASPGHAN22: History of Pediatric GI & Selected Slides from the William F Balistreri Lecture (Part 2)
• NASPGHAN22: History of Pediatric GI & Selected Slides from the William F Balistreri Lecture (Part 3)
• “The Paramount Health Challenge for Humans in the 21st Century”