1. Hospitalization Burden and Incidence of Krabbe Disease.
- Author
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Ghabash G, Wilkes J, Barney BJ, and Bonkowsky JL
- Subjects
- Child, Preschool, Female, Humans, Incidence, Infant, Infant, Newborn, Male, Retrospective Studies, United States epidemiology, Cost of Illness, Hospitalization statistics & numerical data, Leukodystrophy, Globoid Cell epidemiology, Leukodystrophy, Globoid Cell therapy
- Abstract
Objective: The purpose of our study was to understand the healthcare burden and incidence of Krabbe disease (Krabbe)., Methods: Retrospective analysis of Krabbe patients identified October 1, 2015 through December 31, 2020, ages birth through age 3, evaluated in two national databases. We estimated point prevalence and incidence from year 2016 data., Results: We identified 98 unique Krabbe patients with 736 visits including 260 were inpatient admissions. Total healthcare charges were $51.5 million dollars. We determined a point prevalence of 34 68 Krabbe patients in 2016 ages 0 3 years. This estimates a birth incidence of ~1 in 310,000 live births. Significance: Krabbe disease patients had over $51 million in health care charges and hundreds of hospitalizations. Estimated prevalence and birth incidence is similar to rates observed from newborn screening. Our findings show the tremendous health impacts of Krabbe disease, and provide guidance for efforts in screening and treatment planning.
- Published
- 2022
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