Abstract Number: PB0924
Meeting: ISTH 2021 Congress
Theme: Platelet Disorders, von Willebrand Disease and Thrombotic Microangiopathies » VWF and von Willebrand Factor Disorders - Clinical Conditions
Background: von Willebrand disease (VWD) is associated with multiple bleeding-related complications, which negatively impact quality of life (QoL) and result in high healthcare resource utilization (HCRU) in symptomatic patients. More research is required on VWD burden of illness to inform VWD care.
Aims: To describe bleeding-related complications, QoL, and HCRU in patients with VWD.
Methods: A systematic review (PROSPERO CRD42020197674) of observational human studies conducted between January 1, 2010 and July 1, 2020 was performed using the MEDLINE and Embase databases utilizing keywords encompassing both the population (patients with VWD) and data of interest. Obtained studies were screened for eligibility by 2 independent reviewers using predefined criteria. Additional pragmatic searches were performed (Google, Google Scholar, Cochrane Library, abstracts from related conferences) and reference lists of retained publications were screened. Case reports and interventional clinical trials were excluded. For studies with multiple publications, only the most recent was retained. Sources were evaluated and data from relevant publications were extracted.
Results: Within the 122 sources retained, 18 distinct studies described bleeding events. The proportion of patients with bleeding events varied according to bleeding type (Table 1) and VWD severity, with bleeding located primarily in mucocutaneous sites. Eight studies assessing QoL in patients with VWD reported a variety of measures of general health, bodily pain, and physical or social functions. Patients with VWD had poorer QoL scores than reference/general populations in 4 of these 8 studies. One of the 8 studies reporting on HCRU showed approximately 2-fold increases in the rates of outpatient visits and hospitalizations, and longer hospital stays in patients with VWD than in age- and sex-matched controls.
| Bleeding type | Studies, n | Regions | Targeted age (number of studies) |
Patients with VWD, n* | Range for % of patients with bleeding events (overall) | Range for % of patients with bleeding events by VWD type | ||
| Type 1 | Type 2 | Type 3 | ||||||
| Epistaxis | 13 | Europe, North America, Asia-Pacific, Middle East, North Africa, Latin America | All ages (8) ≤19 years (5) |
2,295 | 21.8–86.0 | 38.8–52.9 | 43.8–56.6 | 36.8–77.3 |
| Menorrhagia among female patients |
11 | Europe, North America, Asia-Pacific, Middle East | All ages (7) ≤18 years (3) ≥16 years (1) |
1,573 | 48.3–100 | 79.9–83.3 | 66.7–75.6 | 64.7–69.2 |
| Wound related (including minor wounds/cuts) | 7 | Europe, Asia-Pacific, Middle East | All (3) ≥16 years (2) ≤19 years (2) |
2,796 | 38.0–78.9 | 35.0–51.0 | 39.1–43.8 | 54.5 |
| Gastrointestinal | 13 | Europe, North America, Asia-Pacific, Middle East, North Africa, Latin America | All (8) ≥16 years (1) ≥18 years (1) <18 years (3) |
19,625† | 1.2–24.0 | 4.0–14.3 | 10.3–33.3 | 4.6–19.2 |
| Joint | 9 | Europe, Asia-Pacific, Middle East, Latin America | All (7) ≥16 years (1) <18 years (1) |
3,475 | 3.8–38.0 | 1.9–22.0 | 8.5–20.6 | 36.6–47.8 |
| Central nervous system | 6 | Europe, Middle East | All (2) <16 years (1) ≥16 years (2) <18 years (1) |
3,223 | 1.0–8.8 | 0.6 | 0.8 | 1.8–9.1 |
| Spontaneous oral and dental | 12 | Europe, North America, Asia-Pacific, Middle East, North Africa, Latin America | All (7) ≥16 years (1) ≤18 years (4) |
2,750 | Bleeding‡: 3.6–100 | 27.0–49.0 | 35.7–56.3 | 57.6–69.9 |
| Post-dental extraction | 8 | Europe, Asia-Pacific, Middle East, North Africa, Latin America | All (5) ≥16 years (1) ≤19 years (2) |
2,197 | 6.1–84.6 | 42.0–53.2 | 40.9–60.0 | 40.9 |
| *Patients included in analyses across all studies. †Includes 16,640 admissions from 1 study. ‡Includes oral cavity, gum, and oropharyngeal bleeding. | ||||||||
Proportions of patients with bleeding events reported in observational studies (January 2010 to July 2020)
Conclusions: This literature review highlights the high rates of bleeding-related complications, decreased QoL, and increased HCRU in patients with VWD. Large variations in bleeding rates reflect the variability in study populations and methodologies of the retrieved publications.
To cite this abstract in AMA style:
Du P, Bergamasco A, Arredondo-Bisono T, Moride Y, Truong Berthoz F, Özen G, Tzivelekis S. Burden of Illness in Patients with von Willebrand Disease: A Systematic Review [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/burden-of-illness-in-patients-with-von-willebrand-disease-a-systematic-review/. Accessed September 24, 2023.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/burden-of-illness-in-patients-with-von-willebrand-disease-a-systematic-review/