Inpatient Deaths in Pediatric Leukodystrophies.

Background and Objectives: Leukodystrophies are neurogenetic diseases affecting the white matter of the central nervous system. The contributing factors for leukodystrophy mortality are incompletely understood. Our objectives were to characterize inpatient deaths of pediatric leukodystrophies, including demographics and risk factors. Methods: This retrospective cohort analysis utilized the national Pediatric Health Information System (PHIS) database. Patients under age 19 years with an (ICD-10) diagnosis of leukodystrophy and an inpatient encounter at a PHIS hospital between 2016 and 2024 were included. Analysis included descriptive and multivariate statistics. Results: In all, 613 patients with 15 different leukodystrophies were identified; 32 patients died (5.2%). The most common diagnoses of patients who died were Krabbe disease (KD) (31%), adrenoleukodystrophy (ALD) (28%), metachromatic leukodystrophy (MLD) (28%), and vanishing white matter disease (VWM) (6%). Volume of leukodystrophy admissions at individual hospitals was inversely correlated with mortality. Small numbers limited statistical significance, but mortality rates were higher in females than males (8.3% vs Conclusions: In this large national administrative database study, we characterized and helped define risks for inpatient mortality of pediatric leukodystrophies. Inpatient mortality was correlated with clinical risk factors, and further, although small numbers precluded statistical significance, mortality disparities by sex and race were identified. The reasons for these differences are not known and warrant further study. Our findings identify directions for new research and underscore the potential impact of specialized institutional experience.