Costs of pediatric liver transplantation among commercially- and medicaid-insured patients with cholestasis in the United States.

Children with rare cholestatic liver diseases, such as Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA) typically require liver transplantation (LT). The objective of this analysis was to assess the economic burden of LT on these patients. Healthcare resource utilization (HRU) and costs associated with pediatric LT were retrospectively assessed using insurance claims data from the US IBM MarketScan® Commercial and Medicaid databases collected between October 2015 and December 2019. Inclusion criteria were: ≥1 procedure code for LT, <18 years old at transplant, and ≥6 months of insurance eligibility at baseline. A cholestatic liver disease population who received LT was selected in the absence of specific diagnosis codes by excluding other severe liver conditions (i.e., acute liver failure, malignancy) and by excluding severely decompensated individuals requiring ICU admission before LT. Annualized rates were reported. Over a mean study duration of 1.8 years, 53 commercially- and 100 Medicaid-insured children received LT, with mean (SD) ages at baseline of 6.9 (6.0) and 5.7 (5.4) years, respectively. During this period, commercially- and Medicaid-insured patients had annualized means of 65.3 and 52.8 medical visits, respectively. Most were outpatient visits, although the burden of inpatient visits was also high, with mean inpatient stays (inclusive of LT stay) of 37.2 and 31.6 days per year, respectively. Commercially- and Medicaid-insured patients averaged US$512,124 and $211,863 in medical costs and $26,998 and $15,704 in pharmacy costs, respectively. These costs remained substantial throughout the first-year post-transplant. Overall, pediatric LT resulted in substantial HRU and cost burden in both commercially- and Medicaid-insured patients. Novel targeted medications able to negate the need for pediatric LT could decrease the associated morbidity and costs.