Secondary long-term prophylaxis in von Willebrand disease: an Italian cohort study
Key result
Long-term prophylaxis with factor VIII/VWF concentrates stopped bleeding in 72.7% (8 of 11) of patients with severe von Willebrand disease and reduced hospitalizations in the remaining patients.
Why the study?
Does secondary long-term prophylaxis with factor VIII/VWF concentrates reduce bleeding and hospitalization in patients with severe von Willebrand disease?
Population
452 von Willebrand's disease patients in an Italian cohort, of which 11 patients with severe VWD and…
Design
Cohort
Authors
Discussion
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Implication
May support prophylaxis to reduce bleeding burden in severe von Willebrand disease; hypothesis-generating and leaves open need for randomized confirmation.
Study Design
Type
Cohort (n=452)
Structured PICO
Does secondary long-term prophylaxis with factor VIII/VWF concentrates reduce bleeding and hospitalization in patients with severe von Willebrand disease?
P
Population
Cohort of 452 patients with von Willebrand's disease, of which 11 severe cases with recurrent bleeding received long-term prophylaxis.
E
Exposure
Factor VIII/VWF concentrates (high- or intermediate-purity) 40 FVIII IU/Kg, two-three times a week
O
Outcome
Resolution/reduction of bleeding, numbers of packed red blood cells, and days of hospitalizationhard clinical
Secondary long-term prophylaxis with factor VIII/VWF concentrates appears effective and safe for reducing bleeding and hospitalizations in patients with severe von Willebrand disease.
Limitations
- Cost-effectiveness versus on-demand therapy needs investigation in large prospective studies.
- Lack of large prospective data
- Cost-effectiveness versus on-demand therapy not established
Cite This Study
Federici et al. (2009) conducted a cohort in von Willebrand's disease (VWD) (n=452). Factor VIII/VWF concentrates was evaluated on Resolution/reduction of bleeding, number of packed red blood cells, and days of hospitalization. Long-term prophylaxis with factor VIII/VWF concentrates stopped bleeding in 72.7% (8 of 11) of patients with severe von Willebrand disease and reduced hospitalizations in the remaining patients.