von Willebrand disease (VWD): evidence‐based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA)1

Haemophilia - Tập 14 Số 2 - Trang 171-232 - 2008
William L. Nichols1, MB Hultin2, Allison James3, M. J. Manco‐Johnson4, Robert R. Montgomery5, Thomas L. Ortel6, Margaret E. Rick7, J. Evan Sadler8,9, Mark Weinstein10, Barbara P. Yawn10
1Special Coagulation Laboratory, Division of Hematopathology, Department of Laboratory Medicine and Pathology, and Coagulation Clinic and Comprehensive Hemophilia Center, Division of Hematology and Internal Medicine, College of Medicine, Mayo Clinic, Rochester, MN
2Department of Medicine, Stony Brook University, Stony Brook, NY
3Department of Obstetrics and Gynecology, Duke University Medical Center, Durham, NC
4Department of Pediatrics, University of Colorado Denver, Aurora, CO, and Center for Cancer and Blood Disorders, Children's Hospital of Denver, Denver,CO
5Blood Research Institute, BloodCenter of Wisconsin, and Section of Pediatric Hematology, Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI
6Division of Hematology, Department of Medicine, and Clinical Coagulation Laboratory, Department of Pathology, Duke University Medical Center, Durham, NC
7Hematology Service, Warren Grant Magnuson Clinical Center, National Institutes of Health, Bethesda, MD
8Department of Medicine, Washington University, St. Louis, MO
9§§Office of Blood Research and Review, Center for Biologics Evaluation and Research, US Food and Drug Administration, Rockville, MD
10Department of Research, Olmsted Medical Center, Rochester, MN, and Department of Family and Community Medicine, University of Minnesota, Minneapolis, MN, USA

Tóm tắt

Summary.  von Willebrand disease (VWD) is a commonly encountered inherited bleeding disorder affecting both males and females, causing mucous membrane and skin bleeding symptoms, and bleeding with surgical or other haemostatic challenges. VWD may be disproportionately symptomatic in women of child‐bearing age. It may also occur less frequently as an acquired disorder (acquired von Willebrand syndrome). VWD is caused by deficiency or dysfunction of von Willebrand factor (VWF), a plasma protein that mediates platelet haemostatic function and stabilizes blood coagulation factor VIII. The pathophysiology, classification, diagnosis and management of VWD are relatively complex, but understanding them is important for proper diagnosis and management of patients with VWD. These evidence‐based guidelines for diagnosis and management of VWD from the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel (USA) review relevant publications, summarize current understanding of VWD pathophysiology and classification, and present consensus diagnostic and management recommendations based on analysis of the literature and expert opinion. They also suggest an approach for clinical and laboratory evaluation of individuals with bleeding symptoms, history of bleeding or conditions associated with increased bleeding risk. This document summarizes needs for further research in VWF, VWD and bleeding disorders, including clinical research to obtain more objective information about bleeding symptoms, advancements in diagnostic and therapeutic tools, and enhancement in the education and training of clinicians and scientists in bleeding and thrombotic disorders. The NHLBI Web site (http://www.nhlbi.nih.gov/guidelines/vwd) has a more detailed document, a synopsis of these recommendations, and patient education information.

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