Health-related quality of life in rare bleeding disorders: results from the Rare Bleeding Disorders in the Netherlands study.

ABSTRACT:

Background

Clinical bleeding phenotype varies substantially among patients with rare bleeding disorders (RBDs). Patient-reported outcomes may provide valuable insights into health-related quality of life (HRQoL) and disease burden.

Objectives

To evaluate HRQoL in patients with rare coagulation factor deficiencies and fibrinolytic disorders included in the nationwide, cross-sectional Rare Bleeding Disorders in the Netherlands (RBiN) study.

Methods

Bleeding scores (ie, the International Society on Thrombosis and Haemostasis Bleeding Assessment Tool [ISTH-BAT]) were assessed during a single study visit, and electronic questionnaires captured demographic and HRQoL data (36-item Short Form survey [SF-36], Patient-Reported Outcomes Measurement Information System, Profile 29

SUBMITTER: Willems SPE 

PROVIDER: S-EPMC12304960 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature