Examining variability in the diagnosis and management of people with bleeding disorders of unknown cause: communication from the ISTH SSC Subcommittee on von Willebrand Factor

Abstract

Background: Bleeding disorder of unknown cause (BDUC) is characterised by a bleeding phenotype in the setting of normal haemostatic testing. No standardised diagnostic criteria or treatment algorithms exist for people with BDUC. To address the unmet need, the International Society on Thrombosis and Haemostasis von Willebrand Factor Scientific Subcommittee (ISTH VWF SCC) performed a real-world survey, aimed at addressing knowledge gaps, developing consensus pathways and ultimately improving care. Objectives and Methods: We sought to determine current international clinical practices in the investigation, registration, and treatment of people with BDUC through an online structured survey of health care providers (HCPs) who managed patient with bleeding disorders. Results: Two hundred and sixteen respondents from 39 countries were included in the final analysis. The clinical assessment of those with a possible bleeding disorder varied, with only 55% excluding hypermobility but high levels (80%) of bleeding assessment tools (BAT) usage. In haemostatic testing only the prothrombin time (PT) and activated partial thromboplastin time (APTT) tests gained universal support. Tranexamic acid (TXA) was favoured for prophylaxis for minor (71%)/major (59%) surgeries and pregnancy (58%) but advice on the treatment advised if bleeding occurred was heterogeneous. The management of heavy menstrual bleeding (HMB) in women despite combined oral contraceptive pill (COCP) use also proved challenging with HCPs selecting multiple alternative strategies. Conclusion: Significant variation exists in the recognition, registration and management of people with BDUC worldwide. This survey emphasises the need for consensus pathways to diagnose and treat BDUC to standardise and improve care for patients internationally.