(182) Real-world economic burden of biliary atresia in infants under 1 year of age: A US claims-based analysis

Background: : Biliary atresia (BA) is a rare, progressive pediatric liver disease requiring early intervention to prevent irreversible liver damage. Without timely Kasai portoenterostomy (KP), many patients progress to liver failure, with BA accounting for approximately one-third of pediatric liver transplantations (LT) in the US.

Objective: : This study evaluated the healthcare resource utilization (HCRU) and direct medical costs associated with BA in commercially insured infants in the US who were under 1 year of age and received KP.

Methods: : A retrospective cohort analysis was conducted using Merative™ MarketScan® Commercial Database data from 2016 to 2023. Infants with a first observed diagnostic claim of BA (index date) during their birth year and a claim for KP were included. LT rates and time-to-procedure intervals were assessed using unrestricted follow-up. HCRU and cost outcomes were evaluated descriptively per patient per year (PPPY) within the 12 month post-index period.

Results: : Among 72 infants (53% female), a total of 21 (29%) progressed to LT. The median follow-up was 1.24 years (interquartile range [IQR]: 0.42–3.20) for the KP-only cohort and 2.02 years (IQR: 1.56–3.79) for those who received both KP and LT (KP + LT cohort). Median time from index to KP among infants who underwent KP only (n=51) was 2 days (IQR: 0–7). Among infants who underwent both KP + LT (n=21), the median time from index to KP was 0 days (IQR: 0.0–1.0) and time from KP to LT was 274 days (IQR: 149–367). Compared with the KP only cohort, infants in the KP + LT cohort experienced higher mean numbers of inpatient admissions (3.05 vs 1.73 PPPY) and outpatient visits (64.71 vs 21.94) during the 12 months post-index. The KP + LT cohort also demonstrated modestly higher intensive care unit (1.14 vs 0.45) and emergency room (ER) utilization (2.00 vs 1.04). Median total all-cause costs PPPY were $123,264 (IQR: $49,184–$248,297) for the KP-only cohort and $412,820 (IQR: $101,344–$739,121) for the KP + LT cohort. Median inpatient costs per visit were $61,655 (KP only) and $100,571 (KP + LT). Outpatient and ER per-visit costs were also elevated in the KP + LT cohort.

Conclusions: : This study highlights the substantial burden of care among infants with BA, greatest among those with suboptimal outcomes post-KP leading to LT, and underscores the unmet need for disease-modifying therapies. Additionally, persistently high costs driven by hospitalizations suggest that coordinated care pathways and targeted interventions may improve patient outcomes and reduce economic impact.

Sponsorship:: Ipsen