Assessing the economic burden and health-related quality of life in chinese patients with fibrodysplasia ossificans progressiva: a questionnaire survey analysis.

ABSTRACT:

Background

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, disabling genetic disorder condition that lacks real-world evidence on disease burden. This study aims to investigate the basic characteristics, diagnostic status, prognosis, economic burden and quality of life of FOP patients in China through self-reported data .

Methods

An online survey was conducted through the "China Cloud Platform for Rare Diseases" among patients recruited from the FOP patient organization in March 2023. Patient demographic characteristics, diagnosis, prognosis, healthcare resource usage and costs, and health-related quality of life (HRQoL) data were collected. Health-related quality of life was assessed using EQ-5D-Y for children aged 4-15 and EQ-5D-5L for patients aged ≥ 16. Age

SUBMITTER: Xu X 

PROVIDER: S-EPMC12333074 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature