Patients with Alagille syndrome (ALGS) require many outpatient visits as well as long hospital stays, which contributes to a noteworthy health care resource utilization (HRU) burden, according to a recently published study in The Journal of Pediatrics.

Although ALGS has a relatively low birth prevalence of approximately 1 in every 50,000 births, it is a complex disease. Patients with ALGS often have multiple organ involvement, thus requiring a multidisciplinary team. Furthermore, the majority of children with ALGS will require a liver transplant before the age of 18 years.

“The complexity of care for patients with ALGS is associated with significant healthcare resource utilization (HRU) and economic burden, which impacts patients, their parents, and other caregivers, as well as healthcare systems,” the authors wrote.

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The authors aimed to assess the HRU of ALGS through a retrospective analysis including 386 patients who produced commercial and Medicaid health insurance claims associated with ALGS between 2015 and 2019. The authors used the MarketScan® Commercial Claims and Encounters database and the Multi-State Medicaid database to gather the required data.

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Due to the fact that there is no International Classification of Diseases-10 code for ALGS, researchers used a search algorithm that included claims with the diagnosis code Q44.7 (“other congenital malformations of the liver”) in patients under 18 years of age without a history of biliary atresia. 

More than 60% of the patients included in the study suffered from a cholestatic complication of ALGS. Other common clinical characteristics expressed by the cohort included heart disease (over 30% of patients) and malnutrition (approximately 20% of patients).

Both commercially insured patients and Medicaid-insured patients had more than 20 outpatient visits per year on average. In both groups, laboratory, imaging, and primary care visits were the most frequent types of visits.

There was a significant difference in the length of inpatient visits of both groups; Medicaid patients experienced a mean of 6 inpatient days per year compared to 2.8 inpatient days per year in the privately insured group.

“Improved medical management of ALGS is required to ensure that patients receive optimal and highly subspecialized care, and to reduce the associated economic burden of this condition,” the authors concluded.


Ebel NH, Goldstein A, Howard R, et al. Healthcare resource utilization by patients with Alagille syndromeJ Pediatr. Published online September 26, 2022. doi:10.1016/j.jpeds.2022.09.033