• April 26, 2022
• By admin

Therapeutic targets for hypertrophic cardiomyopathy treatment are continually emerging.

All about hypertrophic cardiomyopathy one must know!

HCM (hypertrophic cardiomyopathy) is a condition in which the heart muscle thickens abnormally (hypertrophied). The heart’s thickened muscle can make it increasingly challenging for it to pump blood. HCM is a type of primary cardiac disease that is passed down the generations as an autosomal dominant feature. Many people with hypertrophic cardiomyopathy go undiagnosed as they have few if any, associated symptoms and can live comfortable lives with no severe complications. Shortness of breath, chest pain, or issues with the heart’s electrical system can occur in a small percentage of people with HCM, leading to life-threatening irregular heart rhythms (arrhythmias) or sudden death. Shortness of breath and heart palpitations can be caused by a variety of conditions. It’s critical to have an early, correct diagnosis and treatment. HCM is classified into two types based on severity. The septum between the two bottom chambers of the heart thickens in obstructive HCM. The pumping chamber’s walls can also grow rigid. It has the potential to obstruct or restrict blood flow from the left ventricle to the aorta. This is the kind that most persons with HCM have. The primary pumping chamber of the heart remains rigid in nonobstructive HCM. The ventricle’s ability to take in and pump out blood is limited, but blood flow is not halted. Echocardiography is usually used to diagnose HCM. It measures the thickness of the heart muscle and the volume of blood flowing through it. Another type of echocardiography, transoesophageal echo (or TEE), may be used in some instances. A TEE is performed with a probe placed into the patient’s throat while they are sedated.

Medical breakthroughs in the treatment of hypertrophic cardiomyopathy.

Although the disease has no remedy, there are a number of treatments that can help to ease the symptoms and improve the quality of life. If you’re aged 30 and also have a parent, child, or sibling with HCM, then the doctor may advise an echocardiography and electrocardiogram every 5 years. If you’re under 30 years old, you’ll be advised to attend more frequently: once every 1-3 years. The most vital medical therapy for patients with HCM is to relieve LV diastolic dysfunction and stimulate regression of LV hypertrophy. Most patients with HCM move from typical HCM to end-stage heart failure when medical therapy fails to improve these two important cardiac diseases. Cibenzoline medication, according to the researchers, may be able to halt the progression of conventional HCM to end-stage heart failure. In patients with HCM, however, preventing abrupt cardiac death is extremely challenging. We cannot change the LV architecture in HCM, which is chaotic and disarrayed cardiac muscle cells with unusual forms, even if the essential cardiac problems in people with HCM may be treated by pharmacological therapy. The most serious issue to be determined is sudden cardiac death in persons with HCM. Existing therapies for hypertrophic cardiomyopathy have been inadequate until now, leaving the majority of patients symptomatic and frequently requiring invasive or even open-heart surgery⁽³⁾.

Molecular treatments that target the underlying mechanisms of Hypertrophic Cardiomyopathy.

Despite the fact that it was initially characterized over 50 years ago, hypertrophic cardiomyopathy still lacks a disease-specific treatment: the medications now in use only relieve symptoms and do not prevent or reverse the phenotype. With recent advances in our understanding of the genetics and pathophysiology of hypertrophic cardiomyopathy, new genetic and pharmacological approaches have been identified and studied that have the potential to act as disease-modifying therapies by influencing different pathways involved in this disease. Our understanding of therapeutic targets for HCM medical therapy is rapidly changing. Novel strategies are being actively pursued to reduce and hopefully prevent the development of severe HCM phenotypes, improve symptom control, and protect patients from disease-related complications, including myocardial metabolic modulation, late sodium current inhibition, and allosteric myosin inhibition. A new treatment on the horizon may be the first to target the fundamental cause of hypertrophic cardiomyopathy. Mavacamten, developed by Bristol-Myers Squibb, has the potential to be a first-in-class medication for treating excessive heart contractions, which can lead to serious illness with restricted blood flow. Bristol Myers Squibb’s new drug application (NDA) for mavacamten, an investigational, innovative oral medicine for patients with symptomatic obstructive hypertrophic cardiomyopathy, was accepted by the FDA in March 2021. The deadline for the FDA to make a decision on the application, known as the PDUFA date, is January 28, 2022. According to interim data from the EXPLORER-HCM extension research, the usage of mavacamten, a cardiac myosin inhibitor, for patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM), showed long-term advantages in quality of life and outcomes. The NDA is based on the findings of phase 3 clinical trial EXPLORER-HCM, which involved 251 patients. Mavacamten had a strong treatment impact in the study, with clinically significant improvements in symptoms, functional status, and quality of life, as well as the ability to reduce left ventricular blockage. All primary and secondary objectives were fulfilled, according to the researchers. The data was published in The Lancet last year⁽⁵⁾.

Reference:

1. Argirò, A., Zampieri, M., Berteotti, M., Marchi, A., Tassetti, L., Zocchi, C., Iannone, L., Bacchi, B., Cappelli, F., Stefàno, P., Marchionni, N., & Olivotto, I. (2021). Emerging Medical Treatment for Hypertrophic Cardiomyopathy. Journal of Clinical Medicine, 10(5), 951.
   https://doi.org/10.3390/jcm10050951
2. Fumagalli, C., De Gregorio, M. G., Zampieri, M., Fedele, E., Tomberli, A., Chiriatti, C., Marchi, A., & Olivotto, I. (2020). Targeted Medical Therapies for Hypertrophic Cardiomyopathy. Current Cardiology Reports, 22(2), 10. https://doi.org/10.1007/s11886-020-1258-x
3. Hamada, M., Ikeda, S., & Shigematsu, Y. (2014). Advances in medical treatment of hypertrophic cardiomyopathy. Journal of Cardiology, 64(1), 1–10. https://doi.org/10.1016/j.jjcc.2014.02.022
4. Hypertrophic Cardiomyopathy (HCM). (n.d.). Www.Heart.Org. Retrieved April 25, 2022, from https://www.heart.org/en/health-topics/cardiomyopathy/what-is-cardiomyopathy-in-adults/hypertrophic-cardiomyopathy
5. Mavacamten May Be a Game-Changing Treatment for Hypertrophic Cardiomyopathy. (n.d.). HCPLive. Retrieved April 25, 2022, from
   https://www.hcplive.com/view/mavacamten-game-changer-hypertrophic-cardiomyopathy
6. New drug shows promise in relieving symptoms associated with hypertrophic cardiomyopathy. (2022, April 3). News-Medical.Net.
   https://www.news-medical.net/news/20220403/New-drug-shows-promise-in-relieving-symptoms-associated-with-hypertrophic-cardiomyopathy.aspx