ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease

• Type of Material: Article
• Authors: Paula D. James, Queens University; Nathan T. Connell, Harvard Medical School; Barbara Ameer, Pharmacology Consulting; Jorge Di Di Paola, Washington University; Jeroen Eikenboom, Leiden University; Nicolas Giraud, Marseille; Sandra Haberichter, Versiti Blood Research Institute; Vicki Jacobs-Pratt, Auburn; Barbara Konkle, Bloodworks Northwest; Claire McLintock, Auckland City Hospital; Simon McRae, Launceston General Hospital; Robert R. Montgomery, Versiti Blood Research Institute; James S. O'Donnell, Royal College of Surgeons in Ireland; Nikole Scappe, Corapolis; Robert Sidonio Jr, Emory University; Veronica Flood, Versiti Blood Research Institute; Nedaa Husainat, University of Kansas; Mohamad Kalot, University of Kansas; Reem A. Mustafa, University of Kansas
• Language: English
• Date: 2021-01-12
• Publisher: ELSEVIER
• Publication Version: Final Published Version
• Copyright Statement: © 2021 by The American Society of Hematology.
• Final Published Version (URL): http://dx.doi.org/10.1182/bloodadvances.2020003265
• Title of Journal or Parent Work: BLOOD ADVANCES
• Volume: 5
• Issue: 1
• Start Page: 280
• End Page: 300
• Keywords: COLLAGEN-BINDING ASSAY; LABORATORY DIAGNOSIS; CLINICAL MARKERS; TYPE-1 VWD; DECISION ETD FRAMEWORKS; BLEEDING ASSESSMENT-TOOL; RISTOCETIN COFACTOR; Science & Technology; INTERNATIONAL NETWORK; FACTOR SURVIVAL; VONWILLEBRAND DISEASE; Life Sciences & Biomedicine; Hematology
• Research Categories: Health Sciences, Medicine and Surgery; Engineering, Biomedical

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.