Abstract Number: PB0952
Meeting: ISTH 2021 Congress
Background: Acquired von Willebrand syndrome (AVWS) is a rare bleeding disorder characterized by a functional or structural defect of von Willebrand factor (VWF) often secondary to autoimmune or lymphoproliferative disorders. Management concentrates on eradicating the autoantibody and achieving hemostasis. Currently, there are no evidence-based guidelines for management, although effectiveness of intravenous immunoglobulin (IVIG) was demonstrated in an open-label crossover study in patients with AVWS.
Aims: Describe a single Center’s experience with management of AVWS.
Methods: Retrospective record review of a 70 y/o Caucasian female diagnosed with AVWS in the setting of Rheumatoid Arthritis (RA) and Parkinson’s disease following recurrent GI bleeding unresponsive to VWF replacement. Coagulation studies suggest severe von Willebrand disease (FVIII 5%, VWF: Ag 11%, VWF R:Co <15%), full gene sequencing of the VWF (exons 1-52) resulted in negative findings, as well as VWF GP1bM and VWF propeptide antigen assay. Suggesting markedly low VWF antigen is due to increased clearance of VWF without inhibition of function secondary to acquired inhibitor, likely resulting from active RA.
Results: IVIG ~1000 mg/kg/dose and high doses of VWF/F8 concentrate achieved normal levels of coagulation proteins for ~1 week, stopping GI bleed. Maintenance low dose IVIG ~1000 mg/kg/dose every 4 weeks has kept patient without bleeding (~10 months), despite maintaining levels of FVIII between 60-20%, VWF: Ag 60-20%, VWF R: Co 30-<15%. Eradication of autoantibody remains unachievable with lack of response to management of underlying disease mechanisms (RA) with Rituximab and Methotrexate.
Conclusions: In patients with AVWS, the use of IVIG and high doses of VWF/F8 concentrate can temporarily increase levels of low proteins to normal range. Favorable response to monthly low dose of IVIG can be given “prophylactically” to prevent bleeding but must consider the risks and benefits of this therapy long-term.
To cite this abstract in AMA style:Escobar M, Chong K, Montanez N. Acquired von Willebrand Disease [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/acquired-von-willebrand-disease/. Accessed September 27, 2023.
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