Experts Put Mavacamten in Perspective

An article published today in Circulation by HCM experts Dr. Steve Ommen of Mayo Clinic and Dr. Martin Maron of Tufts Medical Center, discusses the prospective use of mavacamten as a treatment for obstructive hypertrophic cardiomyopathy. The doctors conclude that while mavacamten (assuming that it is FDA approved in early 2022) will have its place in the HCM tool kit, it should not replace septal reduction therapy for severe HOCM.

In particular, the article points out that the EXPLORER-HCM study showed modest improvements in symptoms and functional capacity (peak V02), comparable to those seen in the RESET-HCM study, which highlighted the ability of regular exercise to improve functional status in HCM.  

The article notes that there has not yet been a study directly comparing mavacamten with septal reduction therapies such as septal myectomy and alcohol septal reduction. The VALOR-HCM study, which is currently recruiting, will look at these therapies compared head-to-head.  It is noteworthy that the majority of patients in the EXPLORER trial had Class II heart failure and were not the more severely compromised Class III and IV patients most likely to benefit from myectomy or alcohol septal ablation.

This article compared historical myectomy data against the findings from EXPLORER, concluding that septal myectomy produces a better result for patients, with gradients abolished in more than 95% of patients compared to only 50% of patients with mavacamten.  And, the article points out that 25% of the patients in the EXPLORER trial continued to have left ventricular outflow tract gradients greater than or equal to 50mmHg, which still qualified them for septal reduction therapy.

Maron and Ommen’s take-home message is that mavacamten will be a welcome addition to the arsenal of HCM drugs and is potentially suitable for patients who do not have severe symptoms, who do not have access to septal reduction at a HCM specialty center, or who wish to avoid more invasive therapies.  It also may be used in the same way as disopyramide, to defer surgery by improving symptoms to a tolerable level.

Lastly, this article points out that there is a need for longer term follow up to study the effects of cardiac remodeling caused by mavacamten.

While it is wonderful to have options, it is important that patients and their medical team consider all available information, including potential benefit and risk, before moving forward with medical therapy. 

Broad Survey of HCM by Dr. Barry J. Maron

The August 16, 2018 online version of the New England Journal of Medicine contains an broad overview of the current state of clinical knowledge and treatment of HCM written by HCM expert Dr. Barry Maron.  It is entitled “Clinical Course and Management of Hypertrophic Cardiomyopathy.” 

Dr. Maron discusses the many advances that have been made in the diagnosis and treatment of hypertrophic cardiomyopathy since it was first described 55 years ago, noting that life expectancy and qualify of life have dramatically improved in this period of time.  According to Dr. Maron, the contemporary management paradigm for HCM have reduced “the risk of adverse cardiovascular events and death to levels below the levels among patients with other cardiac or non-cardiac disorders.”

 

HIGHLIGHTS FROM THE INTERNATIONAL HCM SUMMIT VI, PART III

**Because so much HCM information was presented at the Summit, this is the third of multiple blog entries.  Stay tuned to HCMBeat for more highlights from the HCM Summit.  To see Part I of this series of highlights from the HCM Summit VI, click here and to see Part II of this series click here.**

The symposium was organized by long time HCM expert Dr. Barry Maron and his son, Dr. Martin Maron.  Both Marons are now affiliated with Tufts Medical Center’s Hypertrophic Cardiomyopathy Center.

What follows are summaries from selected talks presented at the meeting.  The presenter and their hospital affiliation are noted below, along with the topic of their presentation.  When possible, you may access the presenters’ slides via hyperlink by clicking on the name. (Note that not all presenters made their slides available).

Continue reading “HIGHLIGHTS FROM THE INTERNATIONAL HCM SUMMIT VI, PART III”

HIGHLIGHTS FROM THE INTERNATIONAL HCM SUMMIT VI, PART II

**Because so much HCM information was presented at the Summit, this is the second of multiple blog entries.  Stay tuned to HCMBeat for more highlights from the HCM Summit.  To see Part I of this series of highlights from the HCM Summit VI, click here.**

The 6th International HCM Summit was held October 27, 28 and 29th in Boston, Massachusetts.  This symposium brings together HCM professionals from around the world who are there to learn about and discuss the latest developments in the treatment of HCM.

The symposium was organized by long time HCM expert Dr. Barry Maron and his son, Dr. Martin Maron.  Both Marons are now affiliated with Tufts Medical Center’s Hypertrophic Cardiomyopathy Center.

What follows are summaries from selected talks presented at the meeting.  The presenter and their hospital affiliation are noted below, along with the topic of their presentation.  When possible, you may access the presenters’ slides via hyperlink by clicking on the name. (Note that not all presenters made their slides available).

Continue reading “HIGHLIGHTS FROM THE INTERNATIONAL HCM SUMMIT VI, PART II”

HIGHLIGHTS FROM THE INTERNATIONAL HCM SUMMIT VI, PART I

**Because so much HCM information was presented at the Summit, this will be the first of multiple blog entries.  Stay tuned to HCMBeat for more highlights from the HCM Summit.  You will find Part II of this series by clicking here.**

The 6th International HCM Summit was held October 27, 28 and 29th in Boston, Massachusetts.  This symposium brings together HCM professionals from around the world who are there to learn about and discuss the latest developments in the treatment of HCM.

The symposium was organized by long time HCM expert Dr. Barry Maron and his son, Dr. Martin Maron.  Both Marons are now affiliated with Tufts Medical Center’s Hypertrophic Cardiomyopathy Center.

What follows are summaries from selected talks presented at the meeting.  The presenter and their hospital affiliation are noted below, along with the topic of their presentation.  When possible, you may access the presenters’ slides via hyperlink by clicking on the name. (Note that not all presenters made their slides available).

Continue reading “HIGHLIGHTS FROM THE INTERNATIONAL HCM SUMMIT VI, PART I”

Outlook Positive for Most HCM Patients

According to an editorial published in yesterday’s issue of Circulation by Drs. Barry and Martin Maron together with Dr. Ethan Rowin, the outlook for most HCM patients is much rosier than previously thought.

According to current estimates, which take into account contemporary disease treatments and risk management strategies, the risk of death from HCM is only .5% per year or 2.5% over 5 years, which is similar to the degree of risk in the general non-HCM population.

Hence, according to the editorial, HCM patients should be reassured about their long term prognosis.