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.

Background: Bleeding disorder of unknown cause (BDUC) is characterized by a bleeding phenotype in the setting of normal hemostatic testing. No standardized 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 performed a real-world survey aimed at addressing knowledge gaps, developing consensus pathways, and ultimately improving care.
Objectives: We sought to determine current international clinical practices in the investigation, registration, and treatment of people with BDUC internationally.
Methods: An online structured survey was conducted of healthcare providers who managed patients with bleeding disorders using the ISTH RedCap tool.
Results: Two hundred 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 tool usage. In hemostatic testing, only the prothrombin time and activated partial thromboplastin time tests gained universal support. Tranexamic acid was favored for prophylaxis for minor (71%)/major (59%) surgeries and pregnancy (58%), but advice on the treatment if bleeding occurred was heterogeneous. The management of heavy menstrual bleeding in women despite combined oral contraceptive pill use also proved challenging, with healthcare providers selecting multiple alternative strategies.
Conclusion: Significant variation exists in the recognition, registration, and management of people with BDUC worldwide. This survey emphasizes the need for consensus pathways to diagnose and treat BDUC to standardize and improve care for patients internationally.
Competing Interests: Declaration of competing interests C.K. has no conflicts to declare. W.T. has served on advisory boards for Grifols, Sanofi, Daiichi Sankyo, Ablynx, Takeda, LFB Biopharmaceuticals, and CSL Behring and received speaker’s fees from Takeda, Bayer, Pfizer, Sobi, Novo Nordisk, AstraZeneca, Alexion, and Portola as well as educational support from CSL Behring, Octapharma, and CSL Behring. M.L. has served on an advisory board for CSL Behring as a consultant for Sobi, CSL Behring, and Band Therapeutics and received speaker fees from Sobi and Takeda. R.I.B.’s institution received grants for clinical research from Bayer, Takeda, Pfizer, Daiichi Sankyo, CSL Behring, Roche, Amgen, Celgene, Rigel Pharmaceuticals, Ionis Pharmaceuticals, AbbVie, Sanofi, MorphoSys AG, Acerta Pharma, Jansen-Cileg, Bristol Myer Squibb, Boehringer Ingelheim, and Astra Zeneca. The institution is in receipt of equipment and reagents for research from Werfen and Technoclone. He received honoraria for speaking at educational meetings from Bayer, Bristol Myers Squibb, Cardinal Health, and Astra Zeneca. He provided expert testimony for Sandoz. He has participated on a Data Safety Monitoring Board or Advisory Boards for Roche, Janssen-Celeg, CSL Behring, Astra Zeneca, and George Institute. J.S.O’D. has served on the speaker’s bureau for Baxter, Bayer, Novo Nordisk, Boehringer Ingelheim, Leo Pharma, Takeda, and Octapharma. He has also served on the advisory boards of Baxter, Bayer, Octapharma CSL Behring, Daiichi Sankyo, Boehringer Ingelheim, Takeda, and Pfizer. J.S.O’D. has served on the speaker’s bureau for Baxter, Bayer, Novo Nordisk, Boehringer Ingelheim, Leo Pharma, Takeda, and Octapharma; has also served on the advisory boards of Baxter, Bayer, Octapharma CSL Behring, Daiichi Sankyo, Boehringer Ingelheim, Takeda, and Pfizer; and has received research grant funding awards from Baxter, Bayer, Pfizer, Shire (now part of Takeda), Takeda, and Novo Nordisk. A.S.L. has no conflicts of interest to declare.
(Copyright © 2024. Published by Elsevier Inc.)