Abstract Number: PB1553
Meeting: ISTH 2020 Congress
Theme: Platelet Disorders and von Willebrand Disease » VWF and von Willebrand Factor Disorders - Clinical Conditions
Background: Von Willebrand disease (VWD) is the most common inherited bleeding disorder with around 0,01% prevalence for the most symptomatic forms where bleeding requires replacement treatment and/or hospitalization.
VWD affects both genders with a higher prevalence and more frequent symptomatic forms in females.
Aims: The aim of the WiSH-QoL study is to characterize the quality of life (QoL) and the impact of the disease and its treatment on daily-life, in VWD patients and their families.
Methods: WiSH-QoL is an observational, ongoing, descriptive, prospective, multicenter study conducted on patients whatever the VWD phenotype, in 28 French centers.
Primary objective is to evaluate VWD patients QoL, using scoring from generic and disease-specific questionnaires, at baseline and at 24 months.
Patients fill QoL questionnaires according to their age: patients ≥18 filled the generic SF-36 and the VWD-QoL; patients aged 4-17 the Disabkids and the VWD-QoL (I or II) questionnaires and their parents filled the corresponding VWD-QoL and the FABEL. For patients < 4, the VWD-QoL 0 and the FABEL questionnaires were filled by parents.
Clinical phenotype such as bleeding Tosetto score is documented.
Results: In this second interim analysis, data at enrolment are presented for 355 patients with a median Tosetto score of 6 [-1; 31].
| n=355 | Type 1 75 (21%) | Type 2 226 (64%) | Type 3 27 (8%) | Unknown 27 (8%) | Total 355 |
| Female | 54 (72%) | 141 (62%) | 14 (52% | 18 (67%) | 227 (64%) |
| Age category (years) <18 | 25 | 76 | 12 | 12 | 125 |
| Age category (years) ≥18 | 50 | 150 | 15 | 15 | 230 |
| Familial VWF deficiency n (%) | 65 (87%) | 178 (79%) | 11 (42%) | 19 (70%) | 273 (77%) |
| Median [range] VWF:RCo (%) | 20 [1; 40] | 13.5 [0.5; 128] | 4 [0; 15] | 15.5 [2.5; 42 | 14 [0; 128] |
| Median [range]VWF:Ag (%) | 22 [3; 55] | 33 [3; 144] | 2 [0.5; 24] | 20.5 [5; 66] | 27 [0.5; 144] |
| Median [range] FVIII (%) | 41 [5; 100] | 42 [2; 202] | 2.5 [1; 57] | 44 [5; 97] | 40 [1; 202] |
| Median [range]Tosetto hemorrhage score | 4 [0; 19] | 7 [-1; 25] | 15.5 [3; 31] | 3 [0; 19] | 6 [-1; 31] |
| On-demand treatment n (%) | 73 (97%) | 222 (98%) | 14 (52%) | 27 (100%) | 336 (95%) |
[Patient’s characteristics at inclusion (n=number of subjects)]
At inclusion, the SF36 questionnaire’s Physical (PCS) and Mental Component Scores (MCS) tended to be more impaired in females: PCS 54.04 vs 55.22; MCS 44.71 vs. 47.85 in females and males respectively.
Conclusions: WiSH-QoL, the first prospective Quality of life study in Von Willebrand disease conducted in France, will provide important insights into a large cohort of type 2 (226/355) patients’ daily-life with their perception of well-being and their specific health care.
These results emphasize too, that all VWD females need a specific healthcare throughout their fertile life.
To cite this abstract in AMA style:
Borel Derlon A, Goudemand J, Deprez D, Volot F, Gruel Y, Chambost H, Veyrardier A, Fressinaud E, Falaise C, Pan-Petech B, von Mackhensen S. WISH-QoL Study: Assessment of Health-Related Quality of Life and Health-Economic Aspects in Patients with von Willebrand Disease in France: Results from the 2nd Interim Analysis on the 355 Enrolled Patients [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/wish-qol-study-assessment-of-health-related-quality-of-life-and-health-economic-aspects-in-patients-with-von-willebrand-disease-in-france-results-from-the-2nd-interim-analysis-on-the-355-enrolled-pa/. Accessed September 27, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/wish-qol-study-assessment-of-health-related-quality-of-life-and-health-economic-aspects-in-patients-with-von-willebrand-disease-in-france-results-from-the-2nd-interim-analysis-on-the-355-enrolled-pa/