A research group of the Cleveland Clinic revealed in recent research that administering an experimental drug, the mavacamtenon patients affected by hypertrophic and severely symptomatic heart disease has significantly reduced the need to resort to invasive therapeutic treatments.
The results of the study “Study Value-HCM: Myosin inhibition as an alternative to surgical myectomy or septal ablation with alcohol in obstructive hypertrophic cardiomyopathy “ were presented to the 71st annual scientific session of the American College of Cardiology in Washington DC.
Mavacamten: this is how the experimental drug works
Research by scientists at the Cleveland Clinic took into account the mavacamten as an alternative to heart surgery or alcohol septal ablation, therapies used to reduce thickening of the septum, the wall that separates the left and right sides of the heart.
Hypertrophic cardiomyopathy is a complex heart disease responsible for thickening the heart, causing stiffness of the left ventricle and causing changes in the mitral valve. This condition affects 600,000 to 1.5 million Americans, or one in 500 people, but many of these patients go undiagnosed until the disease is advanced.
The cause of hypertrophic cardiomyopathy may be unknown or attributed to genetic factors, hypertension, or aging, making it difficult to identify those at high risk of getting it.. Symptoms include chest pain, palpitations, shortness of breath, fatigue, and syncope (fainting).
Most people with hypertrophic cardiomyopathy have a low risk of sudden cardiac death. However, paradoxically, the condition is the most common cause of sudden cardiac death in people under the age of 30.
Medicines such as beta blockers, calcium channel blockers And antiarrhythmics they are often prescribed to treat the symptoms of hypertrophic cardiomyopathy and prevent further complications. Patients with persistent symptoms may also undergo septal myectomy, in which a surgeon removes a small amount of the thickened septal wall to widen the blood outflow tract from the left ventricle to the aorta.
Another option is alcohol ablationa cardiac catheterization procedure in which a small amount of pure alcohol is administered directly into the septum, causing it to shrink to a more normal size and widen the passage for blood flow.
The Phase 3 Valor HCM study involved 112 patients with symptomatic hypertrophic cardiomyopathy at 19 sites in the United States. All patients underwent either surgical myectomy or alcohol ablation. They were randomized to an oral myosin inhibitor, mavacamten (5 to 15 mg per day) or placebo. Mavacamten worked by reducing the excessive contraction of the heart, making it work more efficiently. The drug also reduced the stiffness of the heart muscle.
After 16 weeks, 43 of 56 placebo-treated patients (76.8%) continued to meet the guideline criteria for surgery or chose to undergo surgery compared with 10/56 (17.9% ) patients treated with mavacamten. The study demonstrated a significant reduction in left ventricular outflow tract pressure gradient in patients treated with mavacamten, along with improvements in quality of life measures. The long-term safety and outcomes of mavacamten will continue to be studied.
“These findings could provide what may be a very ill patient population with a non-invasive therapeutic alternative“, he has declared Milind Desaidirector of the Hypertrophic Cardiomyopathy Center and Director of Clinical Operations at the Cleveland Clinic Heart Vascular & Thoracic Instituteand principal investigator of the trial.
“There are few high-volume centers that perform septal myectomy or alcohol ablations that can limit a patient’s access to optimal outcomes, and patients may need repeat surgeries. This is why it is essential to explore non-invasive options for these patients “continued Dessi.
Steven E. Nissen, Chief Academic Officer of the Heart, Vascular & Thoracic Institute at the Cleveland Clinic and senior author of the studies, concluded: “The vast majority of patients, even those in the placebo group, chose to continue the drug after 16 weeks, suggesting a strong interest in this type of treatment ”.
According to Monza Polycliniccenter of excellence for the treatment of hypertrophic cardiomyopathy: “Hypertrophic cardiomyopathy is a disease of genetic origin. It is a disease that leads to the thickening of the heart muscle, which gradually increases in thickness. This thickening can be localized only in part of the left ventricle. Hypertrophic cardiomyopathy is concentric to the ventricle and in particular when it is localized to the septum it can giveobstruction of blood reflux to the aorta which is the main vessel that supplies the entire organism“.
Paolo Ferrazzi, cardiac surgeon, Director of the Center for Hypertrophic Heart Disease and Valve Diseases at the Monza Polyclinic since July 2013 and scientific director of the Department of Cardiac Surgery since July 2014was appointed Knight of the Republic for his professional, humanitarian and scientific merits, gave birth to the International Heart School (Bergamo Foundation for Continuing Medical Education ONLUS, www.ihs-bergamo.it), founded by Prof. Lucio Parenzan in 1989.
The school has trained 350 doctors from 52 countries around the world. Among them, 60 participants hold prestigious roles in their home countries: “A great pride for all of us, including teachers, whose goal now is to create branches of the school also in other continents in order to increase the number of students per year, reducing costs”Said Dr. Ferrazzi with satisfaction.
“I feel responsible for the future of this School and its Mission; to date from Slovenia to China, for which the whole eastern part of the globe, does not have a specialist center for Hypertrophic Cardiomyopathy, it is therefore clear how important it is for the International Heart School to train specialized cardiac surgeons able to bring their knowledge into their own Countries of origin which are often developing areas”, Concluded the scientist.
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