Hospitalization Burden and Incidence of Krabbe Disease

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.

Krabbe Disease–To Add or Not to Newborn Screening?

“…While in 2017, the overall incidence of IKD in Illinois was found to be 1 in 250,000 [5], and 1 in 55,161 in Kentucky respectively [6]. However, a retrospective analysis identified 98 patients ages 0-3 between October 1, 2015, and December 31, 2020, and estimated a birth incidence of about 1 in 310,000 live births [7]. Pediatric Health Information System (PHIS) and Kids' Inpatient Database (KID) data sources indicated a disease burden of 736 hospital visits, with each patient averaging about 2.6 hospitalizations and a length of stay of 33 days [7].…”

Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases

“…The families are notified at this point and the diagnosis is then confirmed with additional testing, such as MRI and nerve conduction tests. 4 , 5 , 87 …”

“… 3 A retrospective analysis of KD cases in the United States estimates an incidence of 1 in 310,000 live births. 4 Newborn screening (NBS) data from New York State estimate an incidence of 1 in 394,000 live births. 5 It should be noted that these estimates are from hospitalization and NBS data, respectively, and both of these data collection methods are biased toward identifying infantile KD.…”

Globoid Cell Leukodystrophy (Krabbe Disease): An Update

“…A study estimated that KD patients had over $51 million in health care charges and hundreds of hospitalizations, demonstrating the health impacts of KD on the society. 43 …”