1Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Stanford University, Stanford, CA. Electronic address: email@example.com.
2Mirum Pharmaceuticals, Inc., Foster City, CA.
3Analysis Group, Inc., Boston, MA.
4Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology, and Nutrition, University of California San Francisco (UCSF), San Francisco, CA.
Objective: (s): To assess and characterize healthcare resource utilization (HRU) in children with the rare, genetic, multisystem disorder, Alagille syndrome (ALGS).
Study design: This retrospective analysis reviewed commercially- and Medicaid-insured claims from October 1, 2015 through December 31, 2019 to assess HRU in patients with ALGS. As there is no specific International Classification of Diseases (ICD)-10 code for ALGS, patients were identified using the following algorithm: ≥1 claim with diagnosis code Q44.7 (other congenital malformations of the liver), <18 years of age, with no history of biliary atresia (ICD-10 code: Q44.2) and ≥6 months of insurance eligibility prior to diagnosis. HRU was summarized per patient per year over all available claims post-diagnosis.
Results: 171 commercially-insured and 215 Medicaid-insured patients with ALGS were available for analysis. Annually, commercially-insured and Medicaid-insured patients averaged 31 medical visits (range 1.5-237) and 48 medical visits (range 0.7-690), respectively. The most common visits were outpatient with the majority encompassing lab/imaging and primary care visits (commercially-insured: 21 [range 0.0-183]; Medicaid-insured: 26 [range 0.0-609]). Inpatient visits were the highest driver of costs in both the commercial and Medicaid populations.
Conclusion: Patients with ALGS have a substantial HRU burden driven largely by numerous outpatient visits and costly inpatient stays. Given the complexity and variability of ALGS presentation, patients may benefit from multidisciplinary and subspecialized care.