cover | Hypertrophic Cardiomyopathy in 2021: New Ideas, New Strategies, and (Possible) New Treatment - American College of Cardiology (2023)

cover | Hypertrophic Cardiomyopathy in 2021: New Ideas, New Strategies, and (Possible) New Treatment - American College of Cardiology (1)

Sixty years ago, hypertrophic cardiomyopathy (HCM) was considered a rare condition with an uncertain prognosis and limited treatment options. It is now thought to affect 1 in 500 people in the general population, and its reputation has been altered by therapeutic innovations to prevent sudden death and treat heart failure (HF) that have significantly reduced morbidity and mortality. disease. .

HCM is currently in the spotlight, with many management advancements highlighted in recent HCM guidelines and, barring regulatory objections, the first new medical therapy in decades (see mavacamten box).

“With a series of advances in modern cardiovascular therapies applied to HCM over the past two decades, we have made tremendous improvements in longevity and quality of life (QOL) for patients, so that almost all patients currently diagnosed with HCM can expect to achieve normal life expectancy with a good quality of life,” he saysMartin S. Maron, dr. z, director of the HCM Center at Tufts Medical Center in Boston, MA, in an interview with Cardiology.

Medical Story or Netflix Drama?

Advanced HCM management

Although hypertrophic cardiomyopathy (HCM) is one of the most common genetic diseases of the cardiovascular system, it is often underdiagnosed, especially in women and in underrepresented populations, and patients suffer from a significant disease burden. With advances in treatment options and new therapies on the horizon, the ACC hosted its first HCM-focused Heart House Roundtable last month to identify and address opportunities and challenges in providing guideline-focused care, and to define the future patient. targeted innovation.

Topics addressed by the group included current standards and limitations in diagnosis and treatment, including multimodal diagnosis, treatment thresholds and interactions, and the overlap between non-obstructive HCM and heart failure with preserved ejection fraction. population health, including access to specialist care, opportunities for value-based models in HCM, and gender and race intersection in HCM.

“HCM covers many fields of medicine, from primary care to cardiology, especially heart failure, to imaging, and requires a full healthcare team, including pharmacists, nurses and others,” noted one panellist. Indeed, the role of interdisciplinary and cross-system therapy has also been explored.

In particular, participants noted a clear need for greater HCM awareness and clinician training, and better accurate recognition and diagnosis of HCM, thanks in part to better image quality and greater use of AI, as well as improved family screening.

Recognizing that access to specialist care is unfair and changes patient outcomes, targets and appropriate metrics are needed to improve access for all, increase the participation of under-represented patients in clinical trials and outcomes studies, and increase the education of clinicians, including the next generation.

The HCM Patient Roundtable Forum was an important opportunity to hear directly from patients how HCM affects their quality of life and highlighted their concerns about the significant delay in accessing specialist HCM, the desire for greater decision-sharing, the need for education patients and support in terms of the impact on their mental health.

The Round Table and the Patient Forum were held as part of the Faculty's new initiative, Accelerated Innovation for Medical Excellence in Hypertrophic Cardiomyopathy (AIME HCM). “Our goal with this initiative is to increase awareness and understanding of HCM, energize clinicians, and optimize disease management to improve outcomes,” said the ACC president.Dipti Itchhaporia, dr med., FACCat the beginning of the Round Table. the AIME HCM Hub and learn more about the roundtable and forum, and access CardioSmart patient resources and helpful training and clinician tools.

Although first documented in the second half of the 19th century, HCM remained largely unrecognized as a clinical entity until the second half of the 20th century.

After the patient was first reported in 1868, there were several groundbreaking observations that led to the discovery of HCM in the 1960s.Eugene Braunwald, MD, MACC, a major player in the story and widely regarded as the father of HCM.1,2

In 1929, a previously healthy 20-year-old student died suddenly while riding a bicycle. An autopsy showed that he had a "huge heart" with hypertrophy, especially of the left ventricle.3

Then in 1949, William Evans, a London cardiologist, described five patients with idiopathic left ventricular hypertrophy (LVH) from three families.4He called the condition "familial cardiomegaly."

This was followed by an autopsy in 1957, considered the first pivotal series to report HCM morphological findings in eight young adults with "asymmetric hypertrophy" of the heart, seven of whom died suddenly,5and a 1961 paper describing a French-Canadian family from Quebec in which 30 members in five generations inherited HCM in an autosomal dominant fashion.6(It wasn't until 1990 that the first gene responsible for HCM was discovered.)

In 1958, Morrow and Braunwald identified two young men with intra-abdominal pressure gradients who were thought to have an unusual congenital anomaly, membranous subaortic stenosis, but found to have no outflow obstruction at the time of surgery where the heart stopped contracting roads, but had an LVH layout.7

Braunwald detailed the clinical features, natural history, and hemodynamics of the condition, in particular the dynamic nature of the obstruction, the incidence of sudden death, autosomal transmission, and tested the newly developed β-blockers as a medical therapy. At the same time, Morrow developed a myectomy, a surgical procedure to relieve a subaortic obstruction that bears his name (the Morrow procedure).

Their joint efforts bore fruitRoad traffica monograph published in 1964 that provided an initial description of HCM as a unique clinical entity, including its pathophysiology and medical and surgical management.8The monograph consisted of 213 pages and contained 176 figures, 18 tables and 191 references. The NIH team named this new disease idiopathic subaortic hyperplasia.

"These people were like Lewis and Clark - they had no idea what was around the corner, they had no cardiovascular imaging to guide them, and they were able to gather all the evidence of this complex disease in a way that still applies today." . . Maron says.

The myectomy procedure described by Morrow, although further developed and advanced, is still the most commonly used surgical procedure for HCM. Beta-blockers, developed in the 1960s and reported by Braunwald and colleagues as offering hemodynamic and clinical benefits, remain the mainstay of medical therapy.

"The history of HCM is simply fascinating, as exemplified by the advances in echocardiography," he saysMichelle M. Kittleson, MD, FACC, director of graduate studies in heart failure and transplantation and director of heart failure research at the Smidt Heart Institute in Cedars-Sinai, Los Angeles, California. “These cardio giants didn't have echocardiography. They performed a physical examination, then identified hemodynamic correlations with invasive cardiac catheterization, and with that information instituted treatment and surgical myectomy.”

(Video) Hypertrophic Cardiomyopathy: Advanced Treatment Options

"Imagine taking a patient to the operating room for a surgical myectomy based on clinical parameters alone, without actually being able to look at the heart and check its thickness," he adds.

cover | Hypertrophic Cardiomyopathy in 2021: New Ideas, New Strategies, and (Possible) New Treatment - American College of Cardiology (2)

With a number of advances in modern cardiovascular therapies applied to HCM over the past two decades,made significant improvements in the longevity and quality of life (QOL) of patients, so almost all patients currently diagnosed with HCM can expect a normal life expectancy with a good quality of life.

Martin S. Maron, dr. z

In a twist fitting the Netflix series, Morrow himself contracted obstructive HCM at the age of 40.8. The diagnosis was made solely on precordial auscultation and examination of symptoms by his close friend and associate, Braunwald.

Morrow's disease was particularly aggressive - he suffered severe symptoms and eventually died at the age of 63 after a series of strokes.9One of his two daughters and his only son also developed progressive forms of the disease, including cardiac arrhythmias, recurrent syncope, heart transplant (daughter, 51), and subaortic obstruction causing severe symptoms of heart failure (son), which resolved with Tomorrow procedure. A 21-year-old granddaughter has HCM, ending the transmission of the disease for three generations.

The evolution of modern HCM management

HCM Guidance Center: Practice Support

The ACC HCM Guideline Center provides a central repository of tools and resources for clinicians needed for the new ACC/AHA HCM Guidelines.

Among the center's highlights: the HCM Guidelines Made Simple document, available in English, Spanish, Portuguese and Chinese, which outlines the top 10 messages of the guidelines. targeted HCM instructional slide sets; expert comments and case study examples; CME and MOC training opportunities. and more. Also find the CardioSmart patient summary and infographic to increase patient knowledge and commitment to collaborative decision herevisit and bookmark Hub.

Test your knowledge and score with two case studies focused on HCM guidelinesJACC: Case hereon joint decision-making on participation in sport.

visit himHCM Guidance Section forJACC.orgto access the second case as well as additionalJACCJournalistic sources.

Complementing efforts to optimize ACC/AHA guidelines, a new tool forACC.orghas been published to make it easier for users to find the instructions. The new search engine is located herevideo tutorial on how to optimize the use of this new tool.

The2020 ACC/AHA guidelines for the diagnosis and treatment of patients with hypertrophic cardiomyopathyoffers a complete overview of the previous 2011 guidelines.10And while the treatment of HCM, both medically and invasively, has not changed significantly in decades (mavacamten is not included in the new guidelines as it has not yet been approved), management strategies have evolved significantly.

"We're getting better at managing this disease - the risk of sudden cardiac death (SCD), arrhythmias, preventing strokes and reversing the symptoms of treatment-resistant heart failure - and the new guidelines make it all clear," says Maron, who served on the writing committee.

Another guideline author, Kittleson, provided a convincing and comprehensive summary of the changes during the HCM session on ACC.21. (click hereto watch an on-demand session on the instruction channel).

The guideline, he notes, consists of 74 pages and 133 recommendations, but includes "Top 10 Home Messages" to help clinicians assimilate the most important details (available atACC.orgGuidance Center). Here are some messages to take home that are especially important to the general cardiologist.

HCM centers: when to report?

General cardiologists should be comfortable making a diagnosis of HCM based on symptom review, physical examination, baseline ECG, echocardiography, and family history as recommended. They can also make initial treatment recommendations and quickly assess changes in the course of the disease.

"General cardiologists may feel more comfortable referring patients for diagnosis confirmation and genetic testing and counseling, or for advanced treatment decisions, such as considering diaphragm reduction therapy or a defibrillator in some cases as part of the primary prevention of SCD," says Kittleson.

cover | Hypertrophic Cardiomyopathy in 2021: New Ideas, New Strategies, and (Possible) New Treatment - American College of Cardiology (3)

(Video) Hypertrophic Cardiomyopathy: Myosin Inhibitors in Context | Rob Fraser, MD

What has gained importance is one thingejection fraction (EF) less than 50%, presence of LV apical aneurysm and presence of extensive post-gadolinium enhancement, which also highlights the increased importance of the role of MRI in risk stratification. MRI in risk stratification.

Michelle M. Kittleson, MD, FACC

While the 2011 guidelines did not specifically recommend when to be referred, the new guidelines say they should be referred for class 2b ICD decisions, diaphragm-reducing therapies, advanced HF management, and decisions related to intense or competitive sports , pregnancy and pediatric HCM.

SCD risk assessment

HCM Management Pearls of Wisdom

Kittleson, known for her#mačićsonpravilashared some gems about management on TwitterCardiologyreaders.

  • In obstructive HCM, treatment should focus on the patient's symptoms rather than the gradient seen on echocardiography, “because the gradient is dynamic. What you really want to know is whether symptoms improve with treatment."
  • If a patient has any component of obstructive HCM, advanced therapies such as heart transplants and LVADs are never indicated because, by definition, obstructive HCM can be treated with medications or invasive therapies to reduce the diaphragm.
  • Traditionally, in HF with reduced EF, the LVEF threshold is <40%. However, for HCM, an EF <50% is considered heart failure and should prompt discussion of ICD, cardiac resynchronization therapy, and guideline medical therapy, as an EF <50% indicates severely reduced systolic function and identifies individuals with a poor prognosis.
  • Don't be fooled by "normal" EF. Some patients develop restrictive physiology and severe symptoms without a decrease in EF. These patients are still sick enough to need a transplant. So don't just use EF as a guide.

SCD remains a devastating consequence of HCM, which makes risk stratification and patient selection important for ICD therapy (Fig.). But the story is completely different from a few years ago.

“Risk stratification in HCM has matured to the point where today we can identify almost all patients at risk for SCD and provide them with ICD protection. It's a remarkable statement and light years from not so long ago when he gave a patient a beta-blocker and hoped you wouldn't get a call that he died," says Maron.

"We used to put everyone with HCM on a treadmill to see if their blood pressure went down during exercise because that was the finding that signaled the need for a defibrillator," says Kittleson.

Studies have shown that it is not an independent risk factor for SCD, nor is the degree of left ventricular outflow tract obstruction and the presence of genetic mutations, he says.

“What has gained importance is an ejection fraction (EF) below 50%, the presence of an apical left ventricular aneurysm, and the presence of extensive gadolinium enhancement,” says Kittleson, who also highlights the increased importance of this role. MRI in risk stratification.

Sports and exercise

Another important change in the new directive concerns sport and activity. Under the old guidelines, low-intensity aerobic exercise was "reasonable" and "it was an overall restrictive and limited class IIa recommendation with a level of evidence of C," says Kittleson.

The new guidelines take into account studies from the last decade that have shown that sudden death during exercise is generally rare in HCM patients, and a moderate-intensity recreational exercise program is not only safe but recommended for the majority of HCM patients, e.g. general population .

“Encouraging exercise may upset some cardiologists. However, they should feel comfortable telling HCM patients to play doubles tennis or hike. Patients should be limited according to their symptoms and exercise at a pace that suits them,” he says. .

When it comes to participating in competitive sports, you should "absolutely" refer to a comprehensive HCM center and HCM specialist, "because there's a lot of gray area around that and a lot of shared decisions," adds Kittleson.

"It's actually about shared decision-making, which we've talked about more clearly and in detail in the guidelines," he notes.

“The updated guidelines are extremely important to us to place new treatments in context as they emerge. There is a lot of expertise and new data from the latest guidelines on what works and what doesn't. This should be taken into account when thinking about how treatments like mavacamten fit into the picture,” he saysDaniel Jacob, Dr. With, founder and director of the cardiomyopathy program at the Yale School of Medicine and investigator in the mavacamten EXPLORER-HCM study.

The promise that is Mavacamten

Mavacamten is a great novelty in the world of HCM. It is a first-in-class, cardiac-specific myosin inhibitor that reduces the number of available actin-myosin cross-bridges, thereby reducing myocardial hypercontraction, improving symptoms and quality of life for patients with obstructive HCM.1In the phase 3 study EXPLORER-HCM, mavacamten (vs placebo) met its primary endpoint, an increase in peak oxygen consumption (pVO2) of ≥1.5 ml/kg/min and at least one decrease in NYHA class or ≥3; pVO2 increase of 0 mL/kg/min and no NYHA class deterioration.1

In the study group, 37% of patients reached the primary endpoint compared to 17% in the placebo group (difference +19.4%, p=0.0005). More patients with mavacamten had both complete symptom relief (Class I) and an outflow gradient to ≤30 mmHg (20.3% vs. 7.8% for placebo, p=0.0005).

(Video) Risk Stratification and Tailored Management of Hypertrophic Cardiomyopathy:Evidence-based Guidelines

Patients treated with Mavacamten showed a greater reduction in the left ventricular outflow tract (LVOT) after exercise compared to placebo (-36 mmHg, p<0.0001) and a greater increase in pVO2 (+1.4 ml/kg/min, p=0 .0006). Approximately one-third more patients in the mavacamten group improved by at least one NYHA class (p<0.0001), with no evidence of safety or tolerability observed. However, it should also be noted that two-thirds of the patients treated with mavacamten did not reach the study endpoint and 50% of the patients were still limited by class II-III symptoms with relatively little improvement in functional capacity.

cover | Hypertrophic Cardiomyopathy in 2021: New Ideas, New Strategies, and (Possible) New Treatment - American College of Cardiology (5)

Never in the 60-year history of this disease have we had a prospective, randomized, double-blind, placebo-controlled trial of a drug of this magnitude that demonstrated such benefits.

Daniel Jacob, Dr. With

The study was conducted on 251 patients enrolled in 68 clinical centers in 13 countries. Participants had gradient LVOT obstruction ≥50 mmHg and NYHA class II-III symptoms. Randomization was taking mavacamtenor placebo for 30 weeks.

In his virtual presentation of the main results at the ESC 2020 Congress, the project managerIacopo Olivotto, Dr. med, called the find a discovery the community has been working on for 60 years. "Nearly 75% of patients experienced a reduction below the guideline thresholds for invasive septal reduction therapy, and 56% showed complete resolution of the obstruction," he said.

It's all in the symptoms

HCM is usually asymptomatic, but once symptoms occur - exercise intolerance, fatigue, angina pectoris, syncope - the impact on quality of life can be profound, and the main goal of treatment is symptom relief.

In a late session of clinical trials at ACC.21,John Spertus, MD, MPH, FACC, presented health outcomes for the EXPLORER-HCM study, with results published concurrently inLanceta.2

At 30 weeks, the change in Kansas City Cardiomyopathy Questionnaire (KCCQ) scores was greater with mavacamten than with placebo (mean scores 14.9 vs. 5.4; difference +9.1, p<0.0001), with similar powers in all KCCQ subscales.

Approximately one in three patients treated with mavacamten (36%) had a ≥20 point change in total sum score compared to 15% in the placebo group, with an estimated absolute difference of 21% and number of cases requiring treatment of five. In the small subgroup with available data, these changes disappeared after discontinuation of treatment.

"Never in the 60-year history of this disease have we had a prospective, randomized, double-blind, placebo-controlled study of a drug of this size show such a large benefit," says Jacoby. He expects the drug to likely be approved.

But apart from that, it's also important to know what the test did and didn't show. “EXPLORER-HCM was not a study for people who needed or were interested in a septal myectomy. It was also not a study directly comparing medical therapy and substrate depletion therapy, so we really need to be careful when comparing these randomized data from EXPLORER-HCM to the retrospective observational data we have on medical treatments and diaphragm reduction,” says Jacoby.

For his part, Maron is less optimistic. “It reduces the contractility of the heart, which reduces the contact time between the mitral valve and the diaphragm, and therefore may reduce outflow tract gradients in many patients with obstruction. Basically, you'd expect it to relieve symptoms, and mavacamten seems to do that in some patients, as we've seen with EXPLORER-HCM," Maron says.

"But what EXPLORER-HCM didn't show was a disease-modifying effect in terms of wall thickness, fibrosis, energy, and so on," he adds. He believes we also need long-term drug data to understand how residual gradients affect long-term efficacy. Maron is the lead researcher forTest REDWOOD-HCM, a phase 2 study of the second-generation myosin inhibitor CK-274 (Cytokinetics), which has a shorter half-life than mavacamten.3The process is still ongoing.

Cost is also an important factor, says Maron. "If this drug is like tafamide, and I expect it to cost more than $80,000 a year or more, and the alternative is a low-risk one-time diaphragm reduction treatment, there's likely to be opposition."

Kittleson takes a cautious but hopeful approach. “Will it replace diaphragm reduction therapy? I don't think so. Will it be an adjunct therapy to β-blockers, calcium channel blockers and disopyramide? Potentially. Will it be good for patients who need diaphragm reduction but for any anatomical reason, weakness or comorbidities are not candidates? Maybe,” he says.

Meanwhile, mavacamten is currently not approved anywhere. In fall 2020, shortly after the presentation of the initial results of the EXPLORER-HCM, Bristol Myers Squibb acquired MyoKardia, the developer of mavacamten, and an application was submitted to the U.S. Food and Drug Administration (PDUFA). ) target date January 28, 2022.

bibliographic references

  1. Olivotto I, Oreziak A, Barriales-Villa R, et al. Mavacamten for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a phase 3 randomized, double-blind, placebo-controlled study.Lanceta2020.; 396: 759-69.
  2. Spertus JA, Fine JT, Elliott P et al. Mavacamten for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a health status analysis in a phase 3 randomized, double-blind, placebo-controlled trial.Lanceta2021? May 14: [Epub ahead of print].
  3. Argiro A, Zampieri M, Berteotti M et al. A new medical therapy for hypertrophic cardiomyopathy.J Clin Med2021.; 10:951.

Fuster's top five influencers (i.e. articles) in HCM

cover | Hypertrophic Cardiomyopathy in 2021: New Ideas, New Strategies, and (Possible) New Treatment - American College of Cardiology (6)

Cardiologyhe askedJACCEditor in ChiefValentin Fuster, MD, MACCto reflect on your opinion on the most important papers published inJournal of American College of Cardiologyover the past five years have helped advance the understanding and treatment of hypertrophic cardiomyopathy (HCM).

(Video) Cardiomyopathy Conference 2020 - Hypertrophic Cardiomyopathy (HCM) - Professor Perry Elliott

Histopfive options are as follows:

cover | Hypertrophic Cardiomyopathy in 2021: New Ideas, New Strategies, and (Possible) New Treatment - American College of Cardiology (7)

Evaluation of Mavacamten in symptomatic patients with non-obstructive hypertrophic cardiomyopathy
Ho CY, Mealiffe ME, Bach RG i in.

the importance of paper?In non-obstructive HCM, which accounts for about half of all HCM cases, treatment options are currently very limited. This study offers some hope.Read the newspaper.

cover | Hypertrophic Cardiomyopathy in 2021: New Ideas, New Strategies, and (Possible) New Treatment - American College of Cardiology (8)

Different subgroups of hypertrophic cardiomyopathy in the NHLBI HCM Registry
Neubauer S, Colm P, Ho CY and in.

the importance of paper?Description of MRI, genetic, and biomarker findings, with two groups developing from these three variables: the sarcomere mutation group is more likely to have an inverted septal curvature morphology, more fibrosis, but less obstruction at rest. and the sarcomere-negative group is more likely to have isolated basal septal hyperplasia, obstruction, but less fibrosis. Only long-term follow-up can tell us the risk stratification and/or prognosis for these two groups.Read the newspaper.

cover | Hypertrophic Cardiomyopathy in 2021: New Ideas, New Strategies, and (Possible) New Treatment - American College of Cardiology (9)

Penetrating hypertrophic cardiomyopathy in sarcomere protein mutation carriers
Lorenzini M, Norrish G, Pole E i in.

the importance of paper?The study, combined with other research by other research groups around the world over three decades, suggests that HCM penetration is likely determined by a number of environmental and genetic influences in addition to the primary sarcomere variant causing the disease. The message from this study is that 1) genetic testing should be performed on patients with HCM and, depending on the results, in first-degree families, and 2) because the disease can appear in early childhood, there may be a need for clinical screening to start at a younger age.Read the newspaper.

cover | Hypertrophic Cardiomyopathy in 2021: New Ideas, New Strategies, and (Possible) New Treatment - American College of Cardiology (10)

Survival after alcohol septal ablation in patients with obstructive hypertrophic cardiomyopathy
Batzner A, Pfeiffer B, Neugebauer A i in.

the importance of paper?For more than 50 years, surgical myectomy has been the gold standard for treating symptomatic patients with high outflow tract gradients, but alcohol septal ablation was introduced as another treatment option more than two decades ago. This study provides long-term follow-up data for patients who have undergone percutaneous septal ablation, which has proven to be a good and safe procedure, with continued improvement in symptoms and excellent long-term survival.Read the newspaper.

cover | Hypertrophic Cardiomyopathy in 2021: New Ideas, New Strategies, and (Possible) New Treatment - American College of Cardiology (11)

Late gadolinium enhancement in patients with hypertrophic cardiomyopathy and preserved systolic function
Mentias A, Raesi-Giglou P, Smedira NG and in.

the importance of paper?This history of sudden cardiac death and risk stratification in HCM patients continues to evolve. This study adds an important variable to such risk stratification - late post-gadolinium enhancement (LGE) on cardiac MRI. This is the first paper to extend the meaning of LGE results to the occlusal and non-occlusive subgroups, as well as to the patient context.Read the newspaper.

(Video) AFCC 2021 - AFCC & ESC Joint Session: Heart Failure Update

bibliographic references

  1. Braunwald E. Hypertrophic cardiomyopathy: the first century 1869-1969.Glob Cardiol Sci Pract2012 .; 2012:5. doi:10.5339/gcsp.2012.5
  2. Naidu SS, ed. Hypertrophic cardiomyopathy. Foreword by Bernard Gersh and historical context by Eugene Braunwald. Springer-Verlag, New York; 2015
  3. Whittle CH. "Idiopathic" myocardial hypertrophy in a young man. Lancet 1929, 213: 1354-55.
  4. Evans W. Familial cardiomegaly.Br Heart J1949, 11:68-82.
  5. Teare D. Asymmetric cardiac hypertrophy in young adults.Br Heart J1958, 20:1-8.
  6. Pare JA, Fraser RG, Pirozynski WJ, Shanks JA, Stubington D. Hereditary cardiovascular dysplasia. A form of familial cardiomyopathy.I am J Med1961, 31:37-62.
  7. Morrow AG, Braunwald E. Functional aortic stenosis? a developmental defect characterized by resistance to left ventricular outflow without anatomical obstruction.Road traffic1959, 20:181-9.
  8. Braunwald E, Lambbrew CT, Rockoff SD, Ross J, Morrow AG. Idiopathic hypertrophic subaortic stenosis. I. Description of the disease based on the analysis of 64 patients.Road traffic1964;30:SUPPL 4:3-119.
  9. Maron BJ, Roberts WC. Father of a septal myectomy for obstructive HCM who also had HCM.J Am Coll Cardiol2016 .; 67:2900-3.
  10. Ommen SR, Mital S, Burke MA et al. 2020 AHA/ACC Guidelines for Diagnosing and Treating Patients with Hypertrophic Cardiomyopathy: Report of the American College of Cardiology/American Society of Cardiology Joint Committee on Clinical Practice Guidelines.J Am Coll Cardiol2020;76:e159-e240.

Keywords: ACC publications,cardiology journal,aneurysm,Heart arythmia,auscultation,Autopsy,benzyloaminy,cycling,Blood pressure,Boston,calcium channel blockers,You have,heart catheterization,cardiac myosins,heart resynchronization therapy,cardiology,cardiomyopathy,hypertrophic cardiomyopathy,Stenosis, pathological,contrast agents,consultation,death, sudden,death, sudden, cardiac,make decisions,defibrylatory,dyzopiramid,double-blind method,echocardiography,electrocardiography,training,ascotherapy,family features,Tiredness,fibrosis,weak old people,friends,gadolin,genetic research,The,half lifeSwoon,heart transplant,heart chambers,hemodynamics,history of medicine,left ventricular hypertrophy,Kansas,Life,London,long life,angels,magnetic resonance imaging,Hermes,mitral valves,Morbidity,Mutation,miozini,nuclear family,operations,oxygen consumption,patient selection,general medical examination,Pregnancy,prescription drugs,primary prevention,Prognosis,prospective studies,quality of life,Quebec,random distribution,referral and consultation,retrospective studies,risk assessment,risk factors,schools, medicine,Social media,stroke,press volumestudents,Swoon,Tennis,uracylbears,abdominal pressure,refusal of treatment,Letter,young adult,AK21,Annual ACC Scientific Session


Are there any new treatments for cardiomyopathy? ›

Mavacamten, a new treatment for hypertrophic cardiomyopathy is changing lives.

When will mavacamten be available? ›

A special message to those outside the USA – additional countries will be seeking authorization of Mavacamten in the coming months or years, and it is anticipated that by 2024 it may be available globally.

What is the last treatment for cardiomyopathy? ›

Septal myectomy is used to treat hypertrophic cardiomyopathy. Heart transplant. A heart transplant might be for people with end-stage heart failure for whom medications and other treatments no longer work.

What is the new drug trial for cardiomyopathy? ›

The drug trials suggest that mavacamten can improve health status in patients with symptomatic hypertrophic cardiomyopathy, including improvements to blood flow, symptoms, well-being and ability to participate in daily activities.

How can I strengthen my heart with cardiomyopathy? ›

Lifestyle Changes for Cardiomyopathy & Heart Failure
  1. Eat Healthfully. Eating a variety of fruits, vegetables, and whole grains and choosing lean meats and fish can help improve your heart health. ...
  2. Stay Active. ...
  3. Monitor Blood Pressure. ...
  4. Monitor Weight. ...
  5. Control Diabetes. ...
  6. Quit Smoking. ...
  7. Limit Alcohol Consumption. ...
  8. Manage Stress.

Has anyone ever recovered from cardiomyopathy? ›

There's no cure for cardiomyopathy. However, you can manage the condition or slow its progression. Many people who make healthy lifestyle choices and seek medical treatment can live a high quality of life with cardiomyopathy.

How much will mavacamten cost? ›

Camzyos Prices, Coupons and Patient Assistance Programs. Camzyos (mavacamten) is a member of the miscellaneous cardiovascular agents drug class and is commonly used for Hypertrophic Cardiomyopathy. The cost for Camzyos oral capsule 2.5 mg is around $8,220 for a supply of 30 capsules, depending on the pharmacy you visit ...

What is the most common treatment for cardiomyopathy? ›

Treatment options for cardiomyopathy
  • Lifestyle changes. Stopping alcohol use. Monitoring salt intake.
  • Medicines. Lower blood pressure. ...
  • Surgically implanted device that helps maintain proper heart rhythm.
  • Ablation procedure. Removes extra heart tissue to reduce thickening. ...
  • Heart transplant (for a severely damaged heart)

What is the new pill for hypertrophic cardiomyopathy? ›

No longer an investigational drug, mavacamten—also known by its brand name, Camzyos—was approved by the U.S. Food and Drug Administration in 2022 for treatment of obstructive HCM. This approval came after a landmark, multicenter clinical trial found it was an effective treatment option for obstructive HCM.

What should you not do with cardiomyopathy? ›

Avoiding alcohol and illegal drugs. Getting enough sleep and rest. Reducing stress. Treating underlying conditions, such as diabetes and high blood pressure.

Which cardiomyopathy has the best prognosis? ›

Patients with peripartum cardiomyopathy appear to have a better prognosis than those with other forms of cardiomyopathy. Patients with cardiomyopathy due to infiltrative myocardial diseases, HIV infection, or doxorubicin therapy have an especially poor prognosis.

What is the best medication for cardiomyopathy? ›

Such medications include beta-blockers, angiotensin-converting enzyme (ACE) inhibitors and angiotensin II receptor blockers (ARBs). Sacubitril/valsartan (Entresto). This drug combines an ARB with another type of medicine to help the heart better pump blood to the rest of the body.

What is the most common drug that causes cardiomyopathy? ›

Although several antipsychotic drugs have been associated with adverse metabolic and cardiovascular effects, only phenothiazines and clozapine have been directly implicated as a primary cause of drug-induced CM.

What is the newest heart treatment? ›

Today, the U.S. Food and Drug Administration approved Farxiga (dapagliflozin) oral tablets for adults with heart failure with reduced ejection fraction to reduce the risk of cardiovascular death and hospitalization for heart failure.

What is the treatment device for cardiomyopathy? ›

Biventricular Pacemaker

The device sends electrical signals to your heart to help it maintain a regular rhythm. Biventricular pacemakers are typically used to manage arrhythmias, or irregular heartbeats, caused by heart failure. This condition causes the organ's lower left chamber, or ventricle, to pump erratically.

Can you get better from cardiomyopathy? ›

Treating cardiomyopathy. There's usually no cure for cardiomyopathy, but the treatments can be effective at controlling symptoms and preventing complications. Some types of cardiomyopathy have specific treatments and early diagnosis is very important.

What foods should I avoid with cardiomyopathy? ›

Avoid cured and processed meats, which are high in sodium. Burgers and steaks, even unseasoned, present their own problem: they're high in the types of fat that can lead to clogged arteries. Instead, aim to eat more fish than red meat, especially salmon, tuna, trout, and cod.

What foods help with cardiomyopathy? ›

Choose foods that are low in salt, such as fresh meats, poultry, fish, dry and fresh legumes, eggs, milk and yogurt. Plain rice, pasta and oatmeal are good low-sodium choices. However, the sodium content can increase if salt or other high-sodium ingredients are added during their preparation.

Which type of cardiomyopathy has poor prognosis? ›

In most cases DCM is progressive, leading to heart failure and death. Without a transplant, the survival rates are poor. DCM has many causes and all of them affect the ventricular function to a varying degree.

Can cardiomyopathy go into remission? ›

Remission of heart failure, defined by resolution of symptoms, normalization of left ventricular ejection fraction, and plasma concentrations of natriuretic peptides and by the ability to withdraw diuretic agents without recurrence of congestion is increasingly recognized among patients with dilated cardiomyopathy.

What is the last stage of cardiomyopathy? ›

In the final stages of heart failure, people feel breathless both during activity and at rest. Persistent coughing or wheezing. This may produce white or pink mucus. The cough may be worse at night or when lying down.

What is a very expensive heart medication? ›

Monitoring Tafamidis, The Most Expensive Cardiac Medication - PMC.

Who owns mavacamten? ›

Mavacamten is a cardiac myosin inhibitor. It was developed by MyoKardia, a subsidiary of Bristol Myers Squibb. Mavacamten was approved for medical use in the United States in April 2022. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.

How is mavacamten administered? ›

Mavacamten is administered orally. Capsules are available in 2.5, 5, 10, and 15 mg doses. The recommended starting dose is 5 mg once a day. Dose titration might occur at weeks 4, 8, and 12 after treatment initiation.

What is the most serious type of cardiomyopathy? ›

Hypertrophic cardiomyopathy

This type of cardiomyopathy causes the heart muscle to become larger and thicker than normal. The thickened areas can block the ventricles (the lower chambers of the heart), making it harder for the heart to pump blood.

Which beta blocker is best for cardiomyopathy? ›

For this reason, carvedilol is usually the first-choice beta blocker for heart failure. Although the long-acting version of metoprolol — metoprolol succinate — has similar benefits to carvedilol. It's important to know that short-acting metoprolol — metoprolol tartrate — may make heart failure worse.

Does metoprolol help cardiomyopathy? ›

CONCLUSIONS. It appears that patients with dilated cardiomyopathy who are treated with metoprolol have enhancement of cell-mediated immunity and improvement of T-cell function; these improvements are correlated to improvement in ejection fraction.

How to prevent sudden death in hypertrophic cardiomyopathy? ›

Ventricular fibrillation is the most common cause of sudden cardiac death (SCD) in hypertrophic cardiomyopathy (HCM). SCD can be prevented by implantable cardioverter defibrillator (ICD) therapy.

What is the new wonder drug for heart failure? ›

Today, the U.S. Food and Drug Administration approved Jardiance (empagliflozin) to reduce the risk of cardiovascular death and hospitalization for heart failure in adults.

What are the chances of dying with cardiomyopathy? ›

It can occur at any age, but it is most shocking when it happens to young adults or athletes. While the media often highlight these tragic deaths, sudden death is rare. It occurs in about 1 out of 100 adults with hypertrophic cardiomyopathy each year.

What triggers cardiomyopathy? ›

Cardiomyopathy can be caused by your gene, other medical conditions, or extreme stress. It can also happen or get worse during pregnancy. Many times, the cause is not known. Treatments include medicines, procedures, and implanted devices.

Can you live a long healthy life with cardiomyopathy? ›

With proper care, many people can live long and full lives with a cardiomyopathy diagnosis. When recommending treatment, we always consider the least invasive approach first. Options range from lifestyle support and medications to implantable devices, procedures, and surgeries.

What is the life expectancy of a person with cardiomyopathy? ›

The majority of patients with hypertrophic cardiomyopathy have no symptoms and most have a near-normal life expectancy. In some cases, sudden cardiac death is the first symptom of the illness. Patients who have symptoms at a younger age often have higher mortality rates.

Can your heart recover from cardiomyopathy? ›

Despite the dramatic presentation, almost all patients recover fully although recurrence rates as high as 5-10% have been reported [15,16]. In-hospital mortality is rare (1.1% in a systematic literature review).

What is the average age of cardiomyopathy? ›

This occurs most often in adults ages 20 to 60. It is more common in men than women, but has been diagnosed in people of all ages, including children. Most people eventually develop heart failure.

Does walking help cardiomyopathy? ›

Walking helps congestive heart failure patients in several ways: Reduces heart attack risk, including cutting the risk of having a second heart attack. Strengthens their hearts and improves lung function. Long term, aerobic activity improves your heart's ability to pump blood to your lungs and throughout your body.

What can I take instead of beta blockers for cardiomyopathy? ›

Ivabradine. Ivabradine is a medicine that can help slow your heart down. It's a useful alternative to beta blockers if you can't take them or they cause troublesome side effects.

Can cardiomyopathy improve with medication? ›

Dilated cardiomyopathy means that your heart can't pump blood around your blood properly. There is no cure, and although it can be treated using common heart medications, there are no treatments specifically for the condition.

What deficiency causes cardiomyopathy? ›

Micronutrients deficiency associated with cardiomyopathy

Although the deficiencies of many micronutrients can cause CM, the most clinically important ones are coenzyme Q10 (CoQ10), thiamine, carnitine, taurine, selenium, and niacin.

What puts you at risk of cardiomyopathy? ›

The major risk factors are: Family history of cardiomyopathy, heart failure or sudden cardiac arrest (SCA) A disease or condition that can lead to cardiomyopathy, such as coronary heart disease, heart attack or a viral infection that inflames the heart muscle. Diabetes or other metabolic diseases, or severe obesity.

What is the number one drug for heart failure? ›

What is the first drug of choice for heart failure? Healthcare providers often prescribe ACE inhibitors and beta blockers as first-line treatments. These drugs are especially helpful for people who have a reduced ejection fraction.

Who makes the number one heart medicine? ›

Top 20 pharma companies by cardiovascular sales
#CompanyGrowth (%)
1Bayer AG10.35%
2Bristol-Myers Squibb Co15.77%
3Johnson & Johnson12.86%
1 more row

What is alternative treatment for cardiomyopathy? ›

Six natural remedies for cardiomyopathy symptoms include eating a heart-healthy diet, controlling contributing conditions (like high blood pressure, high cholesterol and diabetes), exercising and maintaining a healthy weight, sleep and stress management, avoiding alcohol, smoking and illegal drugs as well as natural ...

What drug is Pfizer giving for cardiomyopathy? ›

VYNDAMAX ® (tafamidis) is the first and only, once-daily, single-capsule treatment for both the wild-type and hereditary forms of transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and serious condition.

What is the number one heart medicine in the world? ›

The clear leaders in sales of cardiovascular drugs are Eliquis and Xarelto (NOACs). These two drugs currently represent over 30% of the total cardiovascular market and their shares will continue to expand.

What drugs strengthen the heart muscle? ›

Long term beta blockers help keep your heart failure from becoming worse. Over time, they may also help strengthen your heart. Common beta blockers used for heart failure include carvedilol (Coreg), bisoprolol (Zebeta), and metoprolol (Toprol).

What is the Pfizer heart valve scandal? ›

A unit of Pfizer Inc. has agreed to pay $10.75 million to settle Justice Department claims that the company lied to get Federal approval for a mechanical heart valve that has fractured, killing hundreds of patients worldwide. Under the settlement, which was announced on Thursday, both Pfizer and Shiley Inc.

What is the best COVID vaccine for heart disease? ›

As a heart patient, you should have no concerns about the speed with which the vaccines were developed. The Pfizer-Biontech, Moderna and Johnson & Johnson vaccines were tested on a very large number of patients and shown to be safe and effective.

What is Pfizer's new drug for the heart? ›

VYNDAQEL and VYNDAMAX are indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization.

Is cardiomyopathy a broken heart? ›

Takotsubo cardiomyopathy, also known as broken heart syndrome, is a condition where your heart muscle becomes suddenly weakened, usually because of severe emotional or physical stress.


1. New Data in HFpEF
(CorConsult Rx: Evidence-Based Medicine)
2. 11-4-2021 - Global Cardiovascular Health in the Peri-Pandemic Era
(WashU Med Department of Medicine)
3. Cardiac Sarcoidosis Patient Day Session 2
4. Sarcomere modulation: a new era in management of cardiomyopathies
(Cardiology University of Washington)
5. innovative Cardiology Conference - 5th September 2021
(JAYTECH Live Conferences)
6. VUMC Advanced Heart Failure 2022
(Graduate Medical Education at Vanderbilt Health)


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