ISTH 2023 | Improved quality of life in patients with hemophilia A following gene therapy

So you might think it’s a little bit crazy to measure quality of life if you already know that you can by over 95%, reduce the amount of factor you use and by over 90%, reducing the number of bleeds you have. But it is a very important question. And so they looked at that as in some of the early one, two and three years after gene therapy. And what they found was that there was a significant improvement in quality of life, which I suspect is to a great degree related to those things. But I believe there are also other components which we don’t measure easily. For example, freedom from worry of having a bleed, freedom from thinking about having hemophilia even. I know Glenn Pierce talks about a happiness factor, which I don’t know what that is, but you know, just a sense of less stress as well as reduced bleeds and just going through life with less stress. I think that’s what this points to. It’s a very interesting study and it will be interesting to follow it up longer term. They’re just starting to see some of these changes. So it will be interesting, for example, to see if there’s differences by age, by race, by ethnicity, other things. But so far that’s what we know.