Abstract Number: PB1556
Meeting: ISTH 2020 Congress
Background: The occurrence of anti-von Willebrand Factor inhibitor (anti-VWF) in patients with type 3 von Willebrand disease (3VWD) is a rare but severe complication of the treatment with VWF. It leads to an almost complete therapeutic inefficiency and sometimes to severe allergic reactions.
We report here the off-label use of emicizumab in a 48-year old patient with 3VWD and persistent anti-VWF.
She developed a left ankle arthropathy in 2000 and had a tibiotalar arthrodesis in 2005. She began to experience pain and joint blockings in 2015, which worsened in 2019.
She was currently treated on demand with recombinant Factor VIII (FVIII) infusions (simoctocog alfa, 4,000 IU i.e. 67 IU/kg b.w. per infusion once or twice 2 to 4 hours later due to the short half-life of FVIII in 3VWD patients), but it was less and less effective.
Aims: As emicizumab showed great efficacy on hemophiliac arthropathies, we started an off-label treatment with emicizumab at the end of August 2019 following the recommended dosage.
Methods: Case report.
Results: She experienced a complete relief from pain by the end of the first month of treatment: she has been on emicizumab for 6 months and walks now freely.
We performed Thrombin Generation Assays that showed significant improvement. We also measured the plasmatic concentration of emicizumab: there was no biological sign of neutralizing anti-drug antibody.
Conclusions: Emicizumab showed great efficacy in this patient with left ankle arthropathy. Thus it might be considered in patients with 3VWD and anti-VWF having arthropathies.
To cite this abstract in AMA style:Sigaud M, Fouassier M, Ternisien C, Pennetier M, Trossaert M, Drillaud N. Treatment with Emicizumab Leads to Clinical Improvement in a Type 3 von Willebrand Disease Patient with Inhibitor Suffering from a Left Ankle Arthropathy [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/treatment-with-emicizumab-leads-to-clinical-improvement-in-a-type-3-von-willebrand-disease-patient-with-inhibitor-suffering-from-a-left-ankle-arthropathy/. Accessed October 1, 2023.
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