ross • July 5, 2022
ACC: ORIGINAL INVESTIGATIONS Myosin Inhibition in Patients With Obstructive Hypertrophic Cardiomyopathy Referred for Septal Reduction Therapy

BACKGROUND

Septal reduction therapy (SRT), surgical myectomy, or alcohol ablation, is recommended for obstructive hypertrophic cardiomyopathy (oHCM) patients with intractable symptoms despite maximal medical therapy but is associated with morbidity and mortality.

OBJECTIVES

This study sought to determine whether the oral myosin inhibitor mavacamten enables patients to improve sufficiently to no longer meet guideline criteria or choose to not undergo SRT.

METHODS

Patients with left ventricular (LV) outflow tract (LVOT) gradient$50 mm Hg at rest/provocation who met guideline criteria for SRT were randomized, double-blind, to mavacamten, 5 mg daily, or placebo, titrated up to 15 mg based on LVOT gradient and LV ejection fraction. The primary endpoint was the composite of the proportion of patients proceeding with SRT or who remained guideline-eligible after 16 weeks of treatment.

RESULTS

One hundred and twelve oHCM patients were enrolled, mean age 60 12 years, 51% men, 93% New YorkHeart Association (NYHA) functional class III/IV, with a mean post-exercise LVOT gradient of 84 35.8 mm Hg. After 16 weeks, 43 of 56 placebo patients (76.8%) and 10 of 56 mavacamten patients (17.9%) met guideline criteria or underwent SRT, difference (58.9%; 95% CI: 44.0%-73.9%;P<0.001). Hierarchical testing of secondary outcomes showed significant differences (P<0.001) favoring mavacamten, mean differences in post-exercise peak LVOT gradient 37.2 mm Hg;$1 NYHA functional class improvement 41.1%; improvement in patient-reported outcome 9.4points; and NT-proBNP and cardiac troponin I between-groups geometric mean ratio 0.33 and 0.53.

CONCLUSIONS

In oHCM patients with intractable symptoms, mavacamten significantly reduced the fraction of patients meeting guideline criteria for SRT after 16 weeks. Long-term freedom from SRT remains to be determined.

(A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal ReductionTherapy [VALOR-HCM];NCT04349072)

(J Am Coll Cardiol 2022;80:95–108) © 2022 The Authors. Published by Elsevier on behalf of the American College of Cardiology Foundation.

This is an open-access article under the CC BY-NC-ND license(http://creativecommons.org/licenses/by-nc-nd/4.0/).

For access to the full article please visit : https://www.jacc.org/doi/epdf/10.1016/j.jacc.2022.04.048

Follow-up article by Dr. Steve Ommen: https://www.jacc.org/doi/epdf/10.1016/j.jacc.2022.05.009

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!
An official seal in black & red with white letters that say HCMS Recognized Centers of Excellence

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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