Paula D James1, Nathan T Connell2, Barbara Ameer3,4, Jorge Di Paola5, Jeroen Eikenboom6, Nicolas Giraud7, Sandra Haberichter8, Vicki Jacobs-Pratt9, Barbara Konkle10,11, Claire McLintock12, Simon McRae13, Robert R Montgomery14, James S O'Donnell15, Nikole Scappe16, Robert Sidonio17, Veronica H Flood14,18, Nedaa Husainat19, Mohamad A Kalot19, Reem A Mustafa19. 1. Department of Medicine, Queen's University, Kingston, ON, Canada. 2. Brigham and Women's Hospital, Harvard Medical School, Boston, MA. 3. Pharmacology Consulting, Princeton Junction, NJ. 4. Rutgers-Robert Wood Johnson Medical School, New Brunswick, NJ. 5. Department of Pediatrics, Washington University in St. Louis, St. Louis, MO. 6. Division of Thrombosis and Hemostasis, Department of Internal Medicine, Leiden University Medical Center, Leiden, The Netherlands. 7. Marseille, France. 8. Diagnostic Laboratories, Versiti Blood Research Institute, Milwaukee, WI. 9. Auburn, ME. 10. Bloodworks Northwest, Seattle, WA. 11. Division of Hematology, University of Washington, Seattle, WA. 12. National Women's Health, Auckland City Hospital, Auckland, New Zealand. 13. Northern Cancer Service, Launceston General Hospital, Launceston, TAS, Australia. 14. Versiti Blood Research Institute, Milwaukee, WI. 15. Irish Centre for Vascular Biology, Royal College of Surgeons in Ireland, Dublin, Ireland. 16. Coraopolis, PA. 17. Aflac Cancer and Blood Disorders, Children's Healthcare of Atlanta, Emory University, Atlanta, GA. 18. Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI; and. 19. Outcomes and Implementation Research Unit, Division of Nephrology and Hypertension, Department of Internal Medicine, University of Kansas Medical Center, Kansas City, KS.
Abstract
BACKGROUND: von Willebrand disease (VWD) is the most common inherited bleeding disorder known in humans. Accurate and timely diagnosis presents numerous challenges. OBJECTIVE: These evidence-based guidelines of the American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) are intended to support patients, clinicians, and other health care professionals in their decisions about VWD diagnosis. METHODS: ASH, ISTH, NHF, and WFH established a multidisciplinary guideline panel that included 4 patient representatives and was balanced to minimize potential bias from conflicts of interest. The Outcomes and Implementation Research Unit at the University of Kansas Medical Center (KUMC) supported the guideline-development process, including performing or updating systematic evidence reviews up to 8 January 2020. The panel prioritized clinical questions and outcomes according to their importance for clinicians and patients. The panel used the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, including GRADE Evidence-to-Decision frameworks, to assess evidence and make recommendations, which were subsequently subject to public comment. RESULTS: The panel agreed on 11 recommendations. CONCLUSIONS: Key recommendations of these guidelines include the role of bleeding-assessment tools in the assessment of patients suspected of VWD, diagnostic assays and laboratory cutoffs for type 1 and type 2 VWD, how to approach a type 1 VWD patient with normalized levels over time, and the role of genetic testing vs phenotypic assays for types 2B and 2N. Future critical research priorities are also identified.
BACKGROUND: von Willebrand disease (VWD) is the most common inherited bleeding disorder known in humans. Accurate and timely diagnosis presents numerous challenges. OBJECTIVE: These evidence-based guidelines of the American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) are intended to support patients, clinicians, and other health care professionals in their decisions about VWD diagnosis. METHODS: ASH, ISTH, NHF, and WFH established a multidisciplinary guideline panel that included 4 patient representatives and was balanced to minimize potential bias from conflicts of interest. The Outcomes and Implementation Research Unit at the University of Kansas Medical Center (KUMC) supported the guideline-development process, including performing or updating systematic evidence reviews up to 8 January 2020. The panel prioritized clinical questions and outcomes according to their importance for clinicians and patients. The panel used the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, including GRADE Evidence-to-Decision frameworks, to assess evidence and make recommendations, which were subsequently subject to public comment. RESULTS: The panel agreed on 11 recommendations. CONCLUSIONS: Key recommendations of these guidelines include the role of bleeding-assessment tools in the assessment of patients suspected of VWD, diagnostic assays and laboratory cutoffs for type 1 and type 2 VWD, how to approach a type 1 VWD patient with normalized levels over time, and the role of genetic testing vs phenotypic assays for types 2B and 2N. Future critical research priorities are also identified.
Authors: Gordon H Guyatt; Andrew D Oxman; Regina Kunz; David Atkins; Jan Brozek; Gunn Vist; Philip Alderson; Paul Glasziou; Yngve Falck-Ytter; Holger J Schünemann Journal: J Clin Epidemiol Date: 2010-12-30 Impact factor: 6.437
Authors: Pablo Alonso-Coello; Holger J Schünemann; Jenny Moberg; Romina Brignardello-Petersen; Elie A Akl; Marina Davoli; Shaun Treweek; Reem A Mustafa; Gabriel Rada; Sarah Rosenbaum; Angela Morelli; Gordon H Guyatt; Andrew D Oxman Journal: BMJ Date: 2016-06-28
Authors: J E Sadler; U Budde; J C J Eikenboom; E J Favaloro; F G H Hill; L Holmberg; J Ingerslev; C A Lee; D Lillicrap; P M Mannucci; C Mazurier; D Meyer; W L Nichols; M Nishino; I R Peake; F Rodeghiero; R Schneppenheim; Z M Ruggeri; A Srivastava; R R Montgomery; A B Federici Journal: J Thromb Haemost Date: 2006-08-02 Impact factor: 5.824
Authors: Daniel J Hampshire; Adel M Abuzenadah; Ashley Cartwright; Nawal S Al-Shammari; Rachael E Coyle; Michaela Eckert; Ahlam M Al-Buhairan; Sarah L Messenger; Ulrich Budde; Türkiz Gürsel; Jørgen Ingerslev; Ian R Peake; Anne C Goodeve Journal: Thromb Haemost Date: 2013-05-23 Impact factor: 5.249
Authors: Lynn M Malec; Charity G Moore; Carolyn M Bennett; Donald L Yee; Bryce A Kerlin; Char M Witmer; Roshni Kulkarni; Sweta Gupta; Sriya Gunawardena; Peter A Kouides; Deborah Brown; Margaret V Ragni Journal: J Pediatr Hematol Oncol Date: 2016-03 Impact factor: 1.289
Authors: Carsten Jungbauer; Kurt Debl; Nastasia Roth; Carolin Heidel; Congde Xu; Ute Hubauer; Stefan Wallner; Christine Meindl; Andreas Holzamer; Michael Hilker; Marcus Creutzenberg; Samuel Sossalla; Lars Maier Journal: Clin Res Cardiol Date: 2022-07-15 Impact factor: 6.138
Authors: Stefanie Digiandomenico; Stephen F Conley; Victoria P Johnson; Pamela A Christopherson; Sandra L Haberichter; Jian Zhang; Pippa Simpson; Thomas C Abshire; Robert R Montgomery; Veronica H Flood Journal: Pediatr Blood Cancer Date: 2021-10-04 Impact factor: 3.167
Authors: Dominique P M S M Maas; Ferdows Atiq; Nicole M A Blijlevens; Paul P T Brons; Sandy Krouwel; Britta A P Laros-van Gorkom; Frank W G Leebeek; Laurens Nieuwenhuizen; Selene C M Schoormans; Annet Simons; Daniëlle Meijer; Waander L van Heerde; Saskia E M Schols Journal: J Thromb Haemost Date: 2021-11-21 Impact factor: 16.036