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Gene Therapy for Von Willebrand Disease

par Chuah Lay Khim, Marinee ;Petrus, Inge;VandenDriessche, Thierry
Référence Von Willebrand Disease: Basic and Clinical Aspects, Wiley-Blackwell, page (231-244)
Publication Publié, 2011-03

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Résumé :

An acquired bleeding disorder with clinical and laboratory features similar to inherited von Willebrand disease (VWD) has been reported since 1968 and has been considered to be very rare. It was defined as acquired von Willebrand syndrome (aVWS) because it is similar to VWD and occurs late in life in patients with a negative history of bleeding who suffer mainly from lymphoproliferative, cardiovascular, and myeloproliferative disorders. In most instances aVWS is identified because of bleeding complications, and more than 80% of the patients are active bleeders. New bleeds after the diagnosis has been established occur in about 20% of patients, 33% of whom can require surgery within 1 year of diagnosis. Owing to the high risk of new bleeds and the necessity for surgery in many patients, it is important to establish a firm diagnosis and implement appropriate treatment in these patients. © 2011 Blackwell Publishing Ltd.