ASH 2025 | Emicizumab for severe von Willebrand disease: the EmiVWD study

The EmiVWD study is one that there’s been a lot of desire for in the community. So the hypothesis has been, so for emicizumab or Hemlibra that’s been well used in landscape changing in hemophilia for prophylaxis, could it be used in other bleeding disorders? And so the thought is that, and if you look in the literature, there are patients with severe von Willebrand disease, especially type 3, that have been successfully treated with prophylaxis with emicizumab. But it’s not been studied in a systematic way. And there’s also been questions and anecdotes that other types of variant von Willebrand disease may benefit from the increased thrombin generation that emicizumab can offer. 

So in that regard, we have designed and are enrolling patients on an open-label study where they’re basically doing emicizumab prophylaxis for a year. They’re doing the standard weekly dosing. We chose to do the weekly dosing because the emicizumab levels are the most optimized during that time, as opposed to every twice a month or once a month dosing. We also are allowing for patients not just with severe type 3 von Willebrand disease, but with any variant von Willebrand disease that’s been confirmed either with functional assays or genetically confirmed or even severe type 1s that the clinician feels they have an indication for prophylaxis and have a bleeding history that would be appropriate. Basically, the only patients that we are excluding are those that have mild type 1 von Willebrand disease, so patients in that 30 to 50 range. 

Now, that being said, we also are allowing for patients that have concomitant hemophilia A and von Willebrand disease of any severity. That’s based on the premise that we do have a few patients at our own center that are not on study, but they have severe hemophilia A and also mild von Willebrand disease, have been treated with plasma-derived concentrates in the past, and recombinant factor alone, factor VIII alone, or the combined factor VIII von Willebrand factor concentrates could not optimally control their bleeding. Nevertheless, they were well controlled on emicizumab. So we are allowing for concomitant hemophilia with any severity of von Willebrand disease to be enrolled as well, see what their bleed outcomes are. So per kind of the ASH guidelines, you know, we’re not, we weren’t allowed to give any type of patient enrollment updates, but I’ll say, look for that at a meeting in the near future. So, but we can say that the trial is actively enrolling. We have all of our subsites. It is a US study. And we will look forward to letting the community know how patients are doing once we have the ability to say that.

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