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Prospective evaluation of bleeding phenotype in PT-VWD to support evidence based diagnosis and management

Introduction & Directions


Only 53 patients have been reported with PT-VWD worldwide. Most of these patients; included in the PT-VWD registry; are currently inaccessible with limited follow up. Information about the disease is still lacking with respect to: bleeding phenotype, severity of bleeding symptoms, phenotype-genotype correlations, standardized platelet aggregation based testing and the need for routine DNA analysis for diagnosis. PT-VWD remains under and sometime misdiagnosed. Correct identification is critical to the treatment decision and life-threatening bleeding can occur if not diagnosed/treated appropriately, particularly at times of hemostatic challenges such as surgery, pregnancy and childbirth. Moreover, there is evidence to suggest that abnormalities beyond the bleeding condition may be associated with the abnormally hyper-responsive platelet GPIb alpha. Over the years it has proven extremely difficult to capture the required information about these patients and worldwide cooperation is critical to support evidence-based diagnosis and management.


• To gather detailed information about the clinical and laboratory phenotypes of patients with PT-VWD
• To gather detailed information about the current treatment practices for patients with PT-VWD
• To further understand the treatment challenges and complications with respect to platelet transfusion
• To investigate the bleeding pattern during pregnancy and the occurrence of pregnancy associated complications
• To generate evidence to support a clear diagnostic plan as well as treatment guidelines.