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1mo

At #AHA24, we sat down with Ahmad Masri, MD, of Oregon Health & Science University, about how recent advances in therapy and diagnosis have contributed to the changing risk profile of patients with transthyretin amyloid cardiomyopathy (ATTR-CM) in the US. Learn more about the study: https://lnkd.in/gV4FzucN #Cardiomyopathy

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Yeah, that's a good question. So, you know, we tried to highlight the changing characteristics and severity of the disease, burden of disease. All these patients still have essentially significant amount of disease, still have heart failure. But we started from really the opposite end where patients had extremely advanced disease and now we're bringing it maybe somewhere in the middle. And I think part of the contributions are one, improved recognition of the disease, so you could now diagnose the disease without having to. Without having to do a biopsy, you don't have to wait for the manifestations to be super late before you actually recognize the condition. The use of non invasive diagnosis helped tremendously in this scenario. And then the availability of therapies. But you know, these are the direct contributors. There are a lot of indirect things that are contributing, including the fact that we are taking better care of these patients. When we see someone who has significant better disease, we don't just say, you know, well, there is there are no therapies and I'm sorry, just go home. No, we actually do take care of them. We start them on therapy. We did prescribe. Some of the therapies that are causing their symptoms and you know, you know low blood pressure, frailty as well a contribution to that. We also like you know, enroll patients in exercise programs frequently we do more use more devices in these patients as we can make them live longer. We are treating atrial fibrillation more aggressively in such patients as well compared to what we used in. So I don't think it's one single thing, but all of these are converging at the same time to just simply provide better care overall and make patients live longer. So you're going to end up having to make some choices. It's not like 111 choice only on one-size-fits-all. One is that depending on how the therapy works is either you could enrich the population to be with more disease. That's not always have worked out necessarily well because sometimes the earlier you instituted treatment, the better the outcome could be. But another scenario is totally moving away from fixed time point trials to event driven trials. So an event driven trials. What we mean by that is that we continue the study. And we count how many events we are growing up blinded fashion until we accrue an X amount of events that we part the trial for and then we stop. So of course the downside of that is that the patients have to sign up for a trial that they don't know the duration of. And you have to sign up as an investigator as well for a trial that you don't know what the duration for. So there are trade-offs there. But overall we still see enough events in translation analysis that this is a reasonable design because the duration is now going to be. Extremely long. It might not be exactly 13 months, but it could be 36, it could be 42, it could be, you know, you know, 48. So that's why this event driven trials are useful. And then, you know, finally, I think, you know, selection, it's not always just only tightening the criteria for more sick patients. Sometimes you could essentially, you know, change how you modify these things, like if you're taking patients on therapy, for example, instead of saying any patient can come in, you could consider downloading. Future saying that the patient has to have been, you know, stable on therapy for X amount of time or progressing on therapy for X amount of time and the progression definition is 1-2 and three. So there are ways of getting around it, but I think the primary one is the event driven nature of the trials.

1mo

Thank you Dr. Masri and colleagues for saving lives and outstanding patient care! Your dedication makes a difference!

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