(2024) Current and Emerging PFIC Treatment Landscape

Current and Emerging PFIC Treatment Landscape

2024 PFIC Family & Scientific Conference Family Session

Speaker: Dr. Paula Hertel
Click to read more about Dr. Hertel!


Dr. Hertel is a pediatric gastroenterologist and hepatologist at Texas Children’s Hospital and Associate Professor of Pediatrics at Baylor College of Medicine in Houston, Texas.  Her career is dedicated to caring for children with liver disease and liver transplant, and to clinical research in pediatric liver disease.  She has a particular passion for the care of children with cholestatic liver diseases, including PFIC, and to the study of these complex disorders.  She is a member of the PFIC Writing Group for the National Institutes of Health (NIH) “ChiLDReN” liver research network, and helped to lead this group’s effort in a baseline analysis of children diagnosed with PFIC enrolled in the Network.  At this time, she is working with the PFIC Writing Group on a follow-up study that will include an analysis of serum specimens to determine if lysophosphatidylcholine (LPC) can serve as an effective biomarker (blood test) for itch in children with PFIC.  This work is supported by a PFIC Network Small Research Grant , for which she was a recipient. 

Dr. Hertel is also the Texas Children’s Hospital site’s Principal Investigator for the NIH-sponsored NASH Clinical Research Network, which is focused on study of children and adults with MASLD and MASH (fatty liver disease), and is an ongoing contributor to work published by this group.  She lives in Houston with her husband, two children, pet dog and parrot, and enjoys reading and travel in her free time.  She is extremely grateful to the families and donors of the PFIC Network and thoroughly enjoys the opportunity to participate in the Network’s unique and important collaboration between physicians, researchers, and families.

This topic will explore the latest evidence related to current PFIC treatments and surgical interventions that are most effective in (1) reducing itch related to PFIC and (2) delaying liver transplant. Is there evidence that shows variability per PFIC subtype? Current treatments include antipruritics, bile acid and non-bile acid modulators, other (off-label) treatments, surgical diversions. Additionally, this session will review the latest research on the newest treatment landscape, the IBAT inhibitors. Treatments that are still in clinical trial or pre-clinical phase will be discussed in a later session, Future Treatments of PFIC.

This presentation was recorded at the 2024 PFIC Family & Scientific Conference, which was hosted in partnership with Cincinnati Childrens Hospital. Find out more about PFIC Network Conferences!