title: Changes in bleeding patterns in von Willebrand disease after institution of long-term replacement therapy: results from the von Willebrand Disease Prophylaxis Network.
abstract: Clinically, the leading symptom in von Willebrand disease (VWD) is bleeding, chiefly of mucosal type, for example, epistaxis, gingival, or gastrointestinal bleeding, and menorrhagia. In severe forms of VWD with secondary deficiency of factor VIII, spontaneous joint bleeding, resembling that observed in severe haemophilia A, may also be observed. The bleeding patterns of VWD can affect quality of life, and may be life-threatening. The von Willebrand Disease Prophylaxis Network is an international study group formed with the goal of investigating the role of prophylaxis in clinically severe VWD. The objective of the present study is to investigate the response to prophylaxis focusing primarily on epistaxis, joint bleeding, gastrointestinal bleeding, and heavy bleeding associated with menses. Data from 105 subjects, 10 enrolled in a prospective study and 95 in a retrospective study between 2008 and 2013, were available for analysis. The median annualized rate reductions in bleeding were significant for epistaxis (P < 0.0001), gastrointestinal bleeding (P = 0.0003), joint bleeding (P < 0.0001), and menorrhagia (P = 0.008). Doses on a group level were approximately the same prior to and during prophylaxis, but more patients with gastrointestinal bleeding had prophylaxis three or more times per week as well as higher dosages. Our study, which primarily used retrospective data, indicates that prospective studies are needed to better delineate the doses and dose intervals that should be used for prophylactic treatment of VWD.
authors: aLund University, Malmö Centre for Thrombosis and Haemostasis, Skåne University Hospital, Malmö, Sweden bMedical Sciences Institute, Blood Center of Wisconsin, Milwaukee, Wisconsin cDepartment of Biostatistics, Rho, Inc., Chapel Hill, North Carolina, USA dHaemostasis and Thrombosis Unit, La Paz University Hospital, Madrid, Spain eManchester Blood Centre and the University of Manchester, Manchester, UK fDepartment of Paediatrics, Division of Haematology/Oncology, Hospital for Sick Children, Toronto, Ontario, Canada gAngelo Bianchi Bonomi Hemophilia Thrombosis Centre, IRCCS Ca' Granda Foundation Maggiore Policlinico Hospital and Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy hPediatric Hematology, Medical College of Wisconsin, Comprehensive Center for Bleeding Disorders, Blood Center of Wisconsin, Milwaukee, Wisconsin, USA iGerinnungszentrum Rhein-Ruhr, Duisburg, Germany jAflac Cancer Center and Blood Disorders Service, Children's Healthcare of Atlanta and Emory University, and Department of Hematology and Medical Oncology, Emory University, Atlanta, Georgia kDepartment of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina lDepartment of Medicine, Rochester General Hospital, Rochester, New York, USA mThrombosis and Hemostasis Unit, Rigshospitalet, Copenhagen, Denmark nDepartment of Benigne Hematology Van Creveldkliniek, Erasmus University Medical Center, Rotterdam, The Netherlands oDepartment of Cardiovascular Science, University of Sheffield, Sheffield, UK pAngelo Bianchi Bonomi Hemophilia and Thrombosis Center and Scientific Director of Fondazione IRCCS Cá Granda-Ospedale Maggiore Policlinico, Milan, Italy qUniversity Medical Center Utrecht, Department of Benigne Hematology Van Creveldkliniek, The Netherlands rHematology Research, CHOC Children's Hospital, Orange, California sRush University Medical Center, Chicago, Illinois, USA tCHU Sainte-Justine Hospital, Hemophilia Treatment Centre, Mo
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mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002648:Child; D002675:Child, Preschool; D004844:Epistaxis / Q000150:complications / Q000517:prevention & control*; D005260:Female; D006471:Gastrointestinal Hemorrhage / Q000517:prevention & control*; D006395:Hemarthrosis / Q000150:complications / Q000517:prevention & control*; D006801:Humans; D007223:Infant; D008297:Male; D008595:Menorrhagia / Q000150:complications / Q000517:prevention & control*; D008875:Middle Aged; D011446:Prospective Studies; D012189:Retrospective Studies; D055815:Young Adult; D014842:von Willebrand Diseases / Q000150:complications* / Q000628:therapy*; D014841:von Willebrand Factor / Q000627:therapeutic use*
chemical_list: D014841:von Willebrand Factor