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

Abstract

Background: von Willebrand disease (VWD) is a common inherited bleeding disorder. Significant variability exists in management options offered to patients. Objective: These evidence-based guidelines from 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 health care professionals in their decisions about management of VWD. Methods: ASH, ISTH, NHF, and WFH formed a multidisciplinary guideline panel. Three patient representatives were included. The panel was balanced to minimize potential bias from conflicts of interest. The University of Kansas Outcomes and Implementation Research Unit and the McMaster Grading of Recommendations Assessment, Development and Evaluation (GRADE) Centre supported the guideline development process, including performing and updating systematic evidence reviews (through November 2019). The panel prioritized clinical questions and outcomes according to their importance to clinicians and patients. The panel used the GRADE approach, including GRADE Evidence-to-Decision frameworks, to assess evidence and make recommendations, which were subject to public comment. Results: The panel agreed on 12 recommendations and outlined future research priorities. Conclusions: These guidelines make key recommendations regarding prophylaxis for frequent recurrent bleeding, desmopressin trials to determine therapy, use of antiplatelet agents and anticoagulant therapy, target VWF and factor VIII activity levels for major surgery, strategies to reduce bleeding during minor surgery or invasive procedures, management options for heavy menstrual bleeding, management of VWD in the context of neuraxial anesthesia during labor and delivery, and management in the postpartum setting.

Diagnosis and management of von Willebrand disease: A community‐wide effort to deliver evidence‐based clinical practice guidelines

“…Simultaneously, the National Hemophilia Foundation (NHF) issued a report from their Strategic Summit on VWD that called for ‘A well‐qualified and authoritative organization, or a consortium of such organizations, [to] develop a new or updated evidence‐based clinical practice guideline on VWD’. The American Society of Hematology (ASH) and the International Society on Thrombosis and Haemostasis (ISTH) reached the same conclusions and in 2017 the four organizations came together in an unprecedented international collaboration to develop guidelines on VWD 10,11 …”

“…to develop guidelines on VWD. 10,11 The strongest clinical practice guidelines are developed through a rigorous evidence-based process involving experts in diagnosing, treating and living with a disorder. 12,13 The ASH ISTH NHF WFH Like most clinical practice guidelines, these guidelines face the limitation that they simply cannot cover every topic for which guidance is needed.…”

“…The guideline authors recognize this reality and invite adaptations to local circumstances, based on the associated Evidence-to-Decision frameworks, 17 the details of which are all available in the supplementary materials of the two publications. 10,11 As the community is aware, we lack published prospective studies conducted on large groups of patients with consistently defined outcome assessments and rigorous controls. That the ev-…”

“…The strongest clinical practice guidelines are developed through a rigorous evidence‐based process involving experts in diagnosing, treating and living with a disorder 12,13 . The ASH ISTH NHF WFH 2021 Guidelines on the Diagnosis and Management of VWD are based upon a systematic review and GRADEing of available literature and set a new standard for patient involvement in guideline development 10,11 . While previous VWD guidelines lacked any patient involvement, people with VWD made up approximately a quarter of both the Diagnosis and Management Panels.…”

Phage Display Broadly Identifies Inhibitor-reactive Regions in von Willebrand Factor

“…Abnormalities of VWF result in several types of von Willebrand disease (VWD)[3]. Intravenous infusion of plasma-derived VWF concentrate is the current standard of care for pediatric VWD patients with significant bleeding and poor response to desmopressin[3, 4]. However, the development of anti-VWF alloantibodies (i.e., VWF inhibitors), which primarily occurs in type 3 VWD patients (prevalence of ~1:1,000,000 of the general population), significantly complicates therapy[5, 6].…”