Atrial Fibrillation May Cause Negative Outcomes in HCM

 

A recent study published in Clinical Research in Cardiology  found that atrial fibrillation is a frequent complication of HCM and can be an important predictor of negative outcomes.

These findings suggest that practitioners should aggressively screen for and treat atrial fibrillation in HCM patients to improve long term outcomes.

ESC Issues New Exercise Guidelines Including Guidance for HCM Patients


The European Society of Cardiology recently published new guidelines for exercise for patients with HCM.

These guidelines offer more flexibility for HCM patients that past guidelines, stating that exercise may be permissible for HCM patients, but that it depends on individual situations. The focus should be on the shared decision process between patient and doctor to determine whether exercise may be appropriate for each individual patient.  Additionally, annual follow up for adults, with more frequent follow up for adolescents and young adults is also recommended.

Section 5.5.1 of the guidelines focuses on Hypertrophic Cardiomyopathy. These guidelines state:

  • The risk of sudden cardiac death or arrest (SCD or SCA) during exercise is likely lower than previously thought and therefore, a total ban on exercise is not warranted.
  • Exercise for physical and psychological well-being should be encouraged, following a careful baseline assessment of patients with HCM which should include:
    • Personal and family history
    • Risk Factors for SCD/SCA
    • EKG at rest and during exercise
    • Echocardiogram/Degree of Obstruction
    • Degree of late gadolinium enhancement indicative of cardiac fibrosis as seen on cardiac MRI
    • Exercise testing and blood pressure response to exercise
    • ESC Score of HCM Risk

 

MyoKardia’s EXPLORER Trial Big Success

The results of MyoKardia’s Stage 3 EXPLORER study for its experimental drug mavacamten were presented on Saturday at the European Society of Cardiology’s virtual annual meeting and contemporaneously published in The Lancet

The findings from the study showed that mavacamten improved exercise capacity, reduced left ventricular outflow tract gradient (responsible for the obstruction in HCM) and improved New York Heart Association functional class, correlating with a reduction in symptoms and an improvement in quality of life in patients with obstructive hypertrophic cardiomyopathy. 

This was the largest placebo controlled randomized trial ever conducted in HCM. Additionally, mavacamten is the first drug specifically developed for the treatment of hypertrophic cardiomyopathy. All drugs previously used for HCM symptoms were developed to treat other conditions and were used “off label” to treat HCM.

According to Florence, Italy’s Dr. Iacopo Olivotto, who presented the results to ESC’s virtual audience, mavacamten was generally well tolerated and did not appear to have serious side effects.

MyoKardia said the Explorer-HCM data will form the basis of its submission to the FDA which is planned for the first quarter of 2021.  Last month, the company announced that the drug was awarded “Breakthrough Drug” designation by the FDA which it hopes will streamline the approval process.

Also, a new trial comparing mavacamten to septal myectomy and septal alcohol ablation entitled VALOR-HCM for patients with symptomatic, obstructive HCM and who are New York Heart Association Class III – IV who meet the criteria for septal reduction therapy and have been referred for such a procedure has just gotten underway with the first patient enrolled earlier this month.

DISCLOSURES:  HCMBEAT HAS RECEIVED UNRESTRICTED EDUCATIONAL GRANTS FROM MYOKARDIA.  ADDITIONALLY, CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVED AS A PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL and is credited as an author of the study as published in the lancet.  cynthia also serves on myokardia’s patient advisory board.

Sudden Death Risk Factors Differ for Children with HCM

According to a recent paper published in Circulation, children’s risk for sudden death should be evaluated using different risk factors than those used for adults.

Two main differences seen between factors influencing the risk of sudden death of children and adults were that:

  • for children, family history of sudden death was not a risk factor;
  • a left ventricular outflow tract gradient in a child suggested a lower risk of sudden death.

The authors of this study propose that the risk assessment factors highlighted in this study (and not the various risk assessment tools that have been developed for adults) should be used by clinicians to evaluate the need for implantable defibrillators in children.

 

 

MyoKardia Announces Positive Results from EXPLORER Trial

MyoKardia today announced positive topline results for its experimental drug mavacamten from its recent Stage 3 EXPLORER trial for patients with obstructive hypertrophic cardiomyopathy or HCM.

According to the press release, the drug met all primary and secondary endpoints, while relieving outflow tract obstruction, improving symptoms and enhancing quality of life for the patients enrolled in the trial.  Furthermore, the drug was well tolerated and appeared to be free of significant side effects.

MyoKardia plans to seek FDA approval for mavacamten, the first drug specifically targeting the underlying biological mechanism of HCM, in the first quarter of 2021.

Full results of the EXPLORER study will be presented at a scientific meeting later this year.

 

DISCLOSURES:  CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVES AS PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL AND AS A MEMBER OF MYOKARDIA’S PATIENT ADVISORY BOARD.

 

 

COVID-19: Advice from HCM Doctors

The following guidelines have been developed by HCM specialists at the following hospitals:  Brigham and Women’s Hospital in Boston, Careggi University in Florence, Italy, Stanford University, the University of Michigan and the University of Pennsylvania.

At the bottom of this page you will find the document as a pdf file which should allow you access to the links contained in the document.

Continue reading “COVID-19: Advice from HCM Doctors”

Share Registry Study Focuses on So-Called “End Stage” HCM

Researchers from the Sarcomere Human Cardiomyopathy Registry (or the acronym “SHaRe” for short) have again joined forces to look at a subset of hypertrophic cardiomyopathy (HCM) patients who have what is known as “end stage” HCM.  This term, which is a bit of a misnomer, is used to describe patients who experience left ventricular systolic dysfunction (LVSD) which occurs when the left ventricular ejection fraction (LVEF) is less than 50%.

According to the study published last week in the journal Circulation, of a total of 6,793 HCM patients included in the study, 553 or 8% fell into this category.   The paper goes on to highlight some important observations about patients that fall into this category which are highlighted below.

Continue reading “Share Registry Study Focuses on So-Called “End Stage” HCM”

More Positive Results for MyoKardia Drug

Findings from MyoKardia’s Phase 2 Maverick clinical trial of its experimental drug mavacamten in non-obstructive HCM were announced on Monday at the online 69th Annual Scientific Sessions of the American College of Cardiology.

Dr. Carolyn Ho of Harvard’s Brigham and Women’s Hospital reported to online viewers that mavacamten was generally well tolerated.  In addition, the drug showed promising results in this Phase 2 trial intended to demonstrate the safety of the drug. In particular, some relevant additional observations were reductions in blood levels of two biomarkers of cardiac stress and injury – namely  NT-proBNP which indicates hemodynamic stress, and cardiac troponin I, which is a biomarker of myocardial injury.

These findings look promising for non-obstructive HCM patients who currently have very limited treatment options.

Coronavirus Guidelines for HCM Patients

In consultation with numerous HCM specialists across the U.S., the Hypertrophic Cardiomyopathy Association has compiled a document of guidelines for HCM patients to follow during the current coronavirus crisis.

Some of the specific recommendations are as follows:

  1. Patient with class 3 or 4 heart failure – specifically those with depressed ejection fractions and those awaiting transplantation – should be advised to stay close to home and minimize exposure in social gatherings.
  2. All patients should be encouraged to practice good personal hygiene including frequent hand washing and to use of disinfectants on all surfaces, doorknobs and banisters.
  3. If a HCM patient tests positive for the virus, they should contact their HCM specialist immediately in order to coordinate care with their local physicians and HCM team.

You can find the whole document here.

Additionally, the HCMA has recorded a series of informative webinars with several
HCM experts which you can find here.

Could Artificial Intelligence Be Useful for HCM Screening?

A study by researchers from Mayo Clinic published this week in the Journal of the American College of Cardiology found that an artificial intelligence algorithm was able to detect hypertrophic cardiomyopathy, commonly known as HCM, from EKG results with impressive accuracy, particularly among younger patients.

In order to “teach” the computer, the researchers used digital 12-lead ECGs from 2,448 patients with HCM along with 51,153 age- and sex-matched controls. The technology was then tested on 612 HCM patients and 12,788 controls.

The findings showed that the technology was able to identify HCM in a high number of cases, even where the EKG appeared “normal” to the human eye.

The researchers believe that this technology, when refined, may prove to be an efficient tool for HCM screening in the future. The team plans to continue testing the technology in greater subject samples in order to further refine its performance.

See also:

Mayo Clinic News Network

Health Analytics