Gordon Fox • November 10, 2022
Mavacamten Affects Diastolic Dysfunction

Background

HCM patients commonly suffer from diastolic dysfunction (also called heart failure with preserved ejection fraction (HFpEF), or left-sided heart failure): their hearts do not fill adequately. As a result, even after septal reduction therapy (SRT: myectomy or alcohol septal ablation ), some patients are quite symptomatic. Can mavacamten (Camzyos) help relieve their symptoms? Now a study ( Cremer et al. 2022 ) provides results on how mavacamten affects diastolic dysfunction among patients who have been referred for SRT.

Study design and goals

Researchers randomly assigned patients already on maximal doses of drugs like beta blockers or verapamil to either mavacamten or a placebo. These patients underwent both resting and stress echocardiograms, allowing the researchers to measure diastolic function. The researchers repeated the echos after 16 weeks. They asked several questions: How much did the patients’ diastolic function change? Which parameters from the echos related to changes in diastolic function? What proportion of patients improved in their grade of diastolic dysfunction?

Results

Changes in grades of diastolic dysfunction for patients on mavacamten and placebo.

Fifteen of 51 patients (29%) receiving mavacamten showed improvement in their diastolic dysfunction grade. By contrast, 6 of 47 (13%) of patients in the placebo group improved. Thus mavacamten may have some efficacy in treating diastolic dysfunction. These improvements related to reductions in the E/e’ ratio (an index of the rate of filling in the left ventricle), and to the volume of the left atrium (LAVi) relative to body size. Importantly, the improvements did not depend on changes in the patients’ gradients or their mitral regurgitation.

Looking forward

The improvement in diastolic dysfunction grade is encouraging, suggesting new therapeutic approaches. However, the fact that improvement occurred in fewer than a third of the patients suggests that there is still much to be done in finding more effective treatments.

Literature cited

Cremer, P.C., Geske, J.B., Owens, A., Jaber, W.A., Harb, S.C., Saberi, S., Wang, A., Sherrid, M., Naidu, S.S., Schaff, H.V. and Smedira, N.G., 2022. Myosin Inhibition and Left Ventricular Diastolic Function in Patients with Obstructive Hypertrophic Cardiomyopathy Referred for Septal Reduction Therapy: Insights from the VALOR-HCM Study. Circulation: Cardiovascular Imaging. doi.org/10.1161/CIRCIMAGING.122.014986

HCMA Blog

Lisa's Letter - July Newsletter

By Lisa Salberg • July 3, 2025
Summer, greetings to all our big-hearted friends As July approaches, our focus is already on the fall and preparing for some major events, including our annual meeting coming up in October. July will also find us on the west coast in Seattle conducting a regional patient education meeting as part of our big-hearted warrior tour. We have been following the generic drug quality issue in the United States very closely and encourage you to watch the series starting with our Hill briefing in April and following with the additional webinars with our partners at Medshadow and the People's Pharmacy, and of course the ProPublica series of articles. See them all here . We are happy to have a new team member on board - we welcome Pam as our coordinator of both our All Hearts Collaborative and Hearts and Minds project. Over the next few months, you're going to be learning more about these two amazing initiatives and how we are working to provide better services for big hearts regardless of where you live, so we are meeting all of our big-hearted friends where they are. Please stay tuned for updates from these projects coming soon. We are also creating new volunteer opportunities and engagements that we hope will make it easier for you to participate in spreading the message of the importance of diagnosis, the importance of community readiness related to CPR and AED use and, of course, helping patients get to their ultimate diagnosis and getting them on the proper treatment pathways. This July I would like to recognize all of the special birthdays in my family, including HCMA Center of Excellence coordinator, Stacey Titus-brown and my daughter Rebecca Salberg. It's a milestone birthday for Becca - it’s hard to believe I have a 30-year-old child. Wishing you all a happy and healthy summer. Go build some memories!
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July's Topic is COE Care at the HCMA

By Sabrina Cuddy • July 3, 2025
Getting HCM care at a HCMA Recognized Center of Excellence has a positive impact on patient's outcomes. Find out why in the post by Health Educator Sabrina Cuddy

Pro Publica Releases Results of FDA Investigation

By Erica Friedman • June 26, 2025
Investigative journalists Debbie Cenziper, Megan Rose, Brandon Roberts and Irena Hwang from Pro Publica and NPR have concluded a 14-month long investigation into the quality of generic drugs coming into the United States from overseas. Among the many voices that spoke up for American patients was HCMA Founder and CEO, Lisa Salberg who has felt the effects of low-quality drugs personally. Salberg believes that fixing this problem is something we can do, even in this time, when even health care is highly politicized. Read the key takeaways from ProPublica’s 14-month investigation into the FDA’s oversight of foreign drugmakers in Threat in Your Medicine Cabinet: The FDA’s Gamble on America’s Drugs .
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