Michelle M. Kittleson, MD, PhD, comments on remaining unanswered questions regarding CMIs in treatment of oHCM, highlighting disease modification.
Anjali Owens, MD: Let me take that point over to Michelle. So, Michelle, you said I gave you a softball earlier, but this is the hard one. What is left in terms of unanswered questions, and to build on Milind’s point, what does disease modification mean? You’re a heart failure doctor, like myself . Are we affecting the natural history of disease? What do we need to know to get that answer?
Michelle M. Kittleson, MD, PhD: I think Milind started off the conversation. He punted the volleyball to me. I'm going to spike it over the net with what are the unanswered questions regarding cardiac myosin inhibitors. Like we said, beta blockers are traditionally first-line therapy. But, are they the best first-line therapy? So MAPLE-HCM [hypertrophic cardiomyopathy] is a phase 3 multicenter randomized active comparator clinical trial that's going to pit beta blockers against aficamten in patients with symptomatic obstructive HCM. Looking at a peak VO2 [peak oxygen consumption] change, and I'm very interested to see how that study pans out. I think other things we need to know which everyone's mentioned, I'll just sum up a long-term safety and efficacy cost effectiveness. I think we need to know response based on those important individualized patient traits of sex, race, ethnicity, gene mutation, and then the potential for prevention. So I'm so excited that we have the potential to answer all of these questions.
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