Health-related quality of life in patients with diverse rare diseases: An online survey.

ABSTRACT:

Purpose

Rare diseases substantially contribute to population morbidity and mortality. Understanding rare disease health-related quality of life (HRQL) is essential for evaluating platform-based interventions that aim to tackle multiple rare diseases at a time. However, most HRQL studies focus on single or select group of rare diseases, often in a single country. Our study aimed to identify patient- and disease-specific correlates of HRQL across diverse rare diseases.

Methods

We conducted an international online survey of rare disease patients and caregiver proxies affected by a systematically identified sample of rare diseases. We calculated EQ-5D scores and conducted multivariate linear regression to examine sociodemographic and disease predictors of EQ-5D-5L visual analog

SUBMITTER: Rao A 

PROVIDER: S-EPMC11613748 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature