Patient and family perspectives on lifelong biliary atresia care
Jennifer Lau1,2, Tebyan Rabbani6, Saeed Mohammad3, Alisha Mavis4, Amber Hildreth5, Jennifer Vittorio2, Sanjiv Harpavat7.
1 BARE Inc., Montgomery, IL, United States; 2NYU Langone Health, New York, NY, United States; 3Vanderbilt University Medical Center, Nashville, TN, United States; 4Advocate Health, Charlotte, NC, United States; 5University of California San Diego/Rady Children’s Hospital, San Diego, CA, United States; 6Stanford Medicine Children's Health, Palo Alto, CA, United States; 7Texas Children's Hospital, Houston, TX, United States
Introduction
Biliary atresia (BA) is the leading cause of end-stage liver disease and primary indication of solid organ transplantation in children in the United States1. Patient and family perspectives highlight gaps in BA care, from newborn screening through transplant and transition of care. To address this, we conducted roundtable sessions at the 2025 Cholestatic Liver Disease Summit in Denver, CO2.
Methods
Participants from all cholestatic liver diseases including families, clinicians, researchers and industry partners were engaged in structured roundtable discussions across six domains: pruritus, nutrition, transplant, transition of care, mental health, and Comparative Effectiveness Research (CER)4. Each participant engaged in two topic-specific tables. Real-time qualitative notes were systematically coded using thematic analysis and supplemented with descriptive frequency counts to generate mixed-methods findings2.
Results
BA-specific insights were prominent in Transplant (43%), Transition of Care (36%), and CER (21%) discussions. Transplant-related concerns focused on tacrolimus variability, toxicity, and long-term renal impact, with participants questioning optimal transplant timing in the evolving ileal bile acid transporter (IBAT) inhibitor therapeutic era. Transition challenges were universal, emphasizing inadequate adult-provider preparedness, limited communication, and psychological readiness needed for successful transition3. CER themes highlighted delayed or missed BA diagnoses. Families described inconsistent post-transplant experiences, medication burden, and unmet mental health needs.
Conclusion
As increasing numbers of BA patients reach adulthood, persistent medical, psychosocial, and care-coordination challenges underscore the need for longitudinal, lifespan-oriented care models3. We identified gaps in BA-specific transplant pathways, transition of care infrastructure, and standardized newborn screening. These priority areas should guide future biliary atresia research.

Ipsen . Mirum Pharmaceuticals. Rady Children's Hospital. Rare Disease 360. American Liver Foundation. Medscape. The Forum. EveryLife. Ann & Robert H Lurie Children's Hospital of Chicago. Cincinnati Children's Hospital . Stanford Medicine Children's Health. Sanford Health (CORDS). Children's Organ Transplant Association (COTA). Texas Liver Foundation. UPMC. Kate Farms. Colleens BA 5K. PSC Partners. SPLIT. Healthlink Solutions. Nestle. CF Nutritional Support. Children's Hospital of Philadelphia . Vytala.
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