Health-related quality of life in rare bleeding disorders: results from the Rare Bleeding Disorders in the Netherlands study
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 fac...
Saved in:
| Main Authors: | , , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2025-07-01
|
| Series: | Research and Practice in Thrombosis and Haemostasis |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2475037925002857 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| Summary: | 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 [PROMIS-29]). Only differences exceeding the minimally important difference were considered clinically relevant and reported. Results: HRQoL data from 167 adults and 34 children were available. HRQoL of patients with RBDs measured by SF-36 was not significantly different compared to the Dutch reference population. PROMIS-29 scores indicated significantly better sleep, social participation, and pain-related outcomes in patients with RBDs than the reference populations. Subgroup analyses within the RBiN population showed worse physical health in patients with a severe bleeding phenotype than in those with a mild-to-moderate phenotype. Women with a history of heavy menstrual bleeding reported worse physical health and pain-related outcomes than those without. Patients reporting severe disease had worse pain interference and mental health scores (PROMIS-29) than those reporting nonsevere disease. ISTH-BAT scores were negatively associated with physical functioning. Conclusions: Overall HRQoL in patients with RBDs was comparable to the Dutch reference population. Within the RBiN population, a history of heavy menstrual bleeding, clinical bleeding phenotype, patient-reported disease severity, and ISTH-BAT scores were associated with impaired HRQoL, reflecting disease burden in patients living with RBDs. |
|---|---|
| ISSN: | 2475-0379 |