Home » Hypertrophic cardiomyopathy, towards a new therapy

Hypertrophic cardiomyopathy, towards a new therapy

by admin

There may be a new therapy, the first of a new class, for those suffering from a chronic and progressive heart disease called symptomatic hypertrophic obstructive cardiomyopathy, which affects one in 500 people. The new molecule is called mavacamten and is a potential first inhibitor of myosin which has been shown to improve health conditions compared to placebo at 30 weeks of treatment, as shown by the results of the Explorer-HCM study presented at the 70th annual meeting of the American College of Cardiology’s and published in Lancet.

Hypertrophic cardiomyopathy

But what is hypertrophic cardiomyopathy? A disease in which the heart muscle contracts excessively and in which the filling capacity of the left ventricle decreases, two conditions that can cause heart dysfunction and symptoms such as fatigue and shortness of breath, which can therefore make it difficult to carry out even normal daily activities . Hypertrophic cardiomyopathy is also associated with an increased risk of atrial fibrillation, heart attack, heart failure and sudden cardiac death. The most frequent cause of hypertrophic cardiomyopathy is the mutation of the heart muscle proteins of the sarcomere. Cardiomyopathy is estimated to affect one in 500 people, however in many patients it is undiagnosed or asymptomatic.

I study

The Explorer-HCM study (phase 3, double-blind, randomized and controlled) enrolled a total of 251 patients with symptomatic hypertrophic obstructive cardiomyopathy, who were divided into two groups. Half received the new drug mavacamten and the other half a placebo for 30 weeks, followed by an eight-week washout period. All were asked to complete the Kansas City Cardiomyopathy Questionnaire (KCCQ) – a 23-point questionnaire that quantifies symptoms, physical condition, social function, and quality of life – prior to initiation of therapy and at 6 weeks, 12, 18, 30 and 38. A total of 92 patients who received mavacamten and 88 who received placebo completed the KCCQ questionnaire at baseline and week 30 (end of treatment).

See also  Do they operate under general anesthesia? Be careful if you use the contraceptive pill

At 30 weeks, the overall variation in the questionnaire from the start of the study was greater in patients treated with mavacamten, with a difference of more than 9 points (on average) and with similar benefits across all subscales. The percentage of patients who showed a considerable change (≥20 points) was 36% in the mavacamten vs. 15% in the placebo arm, with an estimated absolute difference of 21%. A change of at least 5 points is required to be considered clinically relevant. These values ​​returned to baseline at the end of active treatment. In addition, a higher proportion of patients in the placebo arm showed no change or deterioration in health status at week 30.

“Using the Kansas City Cardiomyopathy Questionnaire, we can demonstrate the significant clinical benefits for patients treated with mavacamten in the study, which decreased at the end of treatment,” confirms the study’s principal investigator, John A. Spertus, professor of medicine at the University of Missouri – Kansas City and Clinical Director of Outcomes Research at Saint Luke’s Mid America Heart Institute and Lauer Missouri Endowed Chair: “This new data analysis provides important insights into the benefits myosin inhibition can bring in improving patient health. with severe obstructive hypertrophic cardiomyopathy, an often debilitating chronic disease ”.

How the new drug works

Mavacamten is in fact a cardiac myosin modulator, currently being studied for the treatment of conditions that have as fundamental causes the excessive cardiac contractility and the reduced diastolic filling of the heart. The drug reduces the contractility of the heart muscle, inhibiting the excessive bridging between actin and myosin which causes hypercontractility, left ventricular hypertrophy and impaired function. In clinical and preclinical studies, mavacamten consistently reduced heart wall stress biomarkers, excessive cardiac contractility, and increased diastolic function.

See also  Colon cancer: Bailey (24) has to burp constantly – then doctors make a shock diagnosis

.

You may also like

Leave a Comment

This site uses Akismet to reduce spam. Learn how your comment data is processed.

This website uses cookies to improve your experience. We'll assume you're ok with this, but you can opt-out if you wish. Accept Read More

Privacy & Cookies Policy