EXPLORER-HCM Trial shows dramatic improvements in patients with common genetic heart condition, comes after decades of no new treatments

May 19, 2021

Saint Luke’s Mid America Heart Institute announced the results of a clinical trial that will impact patient care and improve quality of life for patients suffering from the most common genetic heart condition, called hypertrophic cardiomyopathy (HCM). This news comes after 30 years of no new treatments for the condition.

Dr. John Spertus presented a detailed analysis of the EXPLORER-HCM trial, based on the Kansas City Cardiomyopathy Questionnaire he developed, for the first time at the virtual American College of Cardiology’s 70th Annual Scientific Session. The ACC’s yearly conference brings together many of its 54,000 members made up of cardiovascular professionals across the globe, where the latest breakthroughs in science and research are presented.

Hypertrophic cardiomyopathy (HCM) is a complex type of heart disease that thickens the heart muscle. It is the most common genetic heart condition but often goes unrecognized. The condition can worsen over time and lead to poor function, quality of life, and long-term complications. People with this condition have a higher risk of developing atrial fibrillation, heart failure, and even sudden cardiac arrest or death. Historically, treatments for HCM have been very limited.

In the phase 3 randomized trial of patients with symptomatic HCM, Spertus and a team of researchers looked at how a new oral medication manufactured by Bristol Myers Squibb, called mavecamten, impacted patients’ quality of life, physical function, and symptoms. The study took place at 86 cardiovascular centers in 13 different countries. People who participated in the trial were randomly assigned to take mavacemten or a placebo pill once a day over 30 weeks and continued to be monitored the next eight weeks after stopping the medication.

Patients who took mavacamten showed significant improvements in their Kansas City Cardiomyopathy Questionnaire scores, which measures patients’ quality of life, function, and symptoms. These patients noted benefits early on after starting treatment, which then dropped off after stopping the medication.

“It is a remarkable advance to the limited resources available to treat these patients,” Dr. Spertus said. “The benefits observed were among the largest we have ever seen from a medication to improve the symptoms, function, and quality of life of patients with heart failure. Finding that these benefits dissipated after stopping medication further supports the direct benefits of treatment and underscores the potential for this new treatment to help patients with obstructive HCM.”

Read the full analysis published in The Lancet.


Saint Luke's Mid America Heart Institute

Saint Luke's Mid America Heart Institute is a member of Saint Luke's Health System, which consists of 16 area hospitals and several primary and specialty care practices, and provides a range of inpatient, outpatient, and home care services. Founded as a faith-based, not-for-profit organization, our mission includes a commitment to the highest levels of excellence in health care and the advancement of medical research and education. The health system is an aligned organization in which the physicians and hospitals assume responsibility for enhancing the physical, mental, and spiritual health of people in the metropolitan Kansas City area and the surrounding region.

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