Significant healthcare burden and life cost of spinal muscular atrophy: real-world data.

ABSTRACT:

Objectives

The aim of this study is to quantify the mortality rate, direct healthcare costs, and cumulative life costs of pediatric patients with spinal muscular atrophy (SMA) type 1, type 2, and type 3 born in Hong Kong.

Methods

Data were collected from genetically confirmed SMA patients born in or after 2000 from the Hospital Authority medical database. Patients were followed up from birth until they died, left Hong Kong, reached 18 years, or initiated disease-modifying treatment. Study outcomes included incidence risks of mortality, cumulative direct medical costs-attendances of special outpatient clinics, emergency department, allied health services, and mean length of stay in hospitals over time. Total direct medical costs were calculated as unit costs multiplied by uti

SUBMITTER: Chan SHS 

PROVIDER: S-EPMC10533630 | biostudies-literature | 2023 Nov

REPOSITORIES: biostudies-literature