The results from the Myokardia EXPLORER- MRI sub-study are in, and there is more positive news for mavacamten (formerly known as MYK-461). According to the results of this small 35 patient study, patients who took mavacamten showed reduction of left ventricular size and wall thickness on MRI. These patients also had a reduction in their left atrial volume. All three of these measurements are predictors of poor outcome for HCM patients Additionally, this study found a reduction in certain biomarkers such as NT-proBNP, which indicate heart stress and injury.
MyoKardia (which was recently acquired by Bristol Myers Squibb) hopes that this drug will be approved by the FDA and available to American HCM patients by the end of this year.
For more on the progress of mavacamten, read these previous entries on HCMBeat:
Positive Results for MyoKardia Drug Mavacamten
MyoKardia Announces Positive Result for Mavacamten for Treatment of HOCM
MyoKardia Announces Positive Results from EXPLORER Trial
More Positive Results for MyoKardia Drug
MyoKardia’s EXPLORER Trial Big Success
DISCLOSURES: CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVED AS A PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL. CYNTHIA ALSO SERVES ON MYOKARDIA’S PATIENT ADVISORY BOARD
According to a recently published study by doctors in Copenhagen, Denmark, myocardial crypts (clefts, cracks or fissures in the myocardium) are found in the general population. Therefore, this article concludes that crypts seen on scans of the heart are not necessarily an indicator of HCM and do not warrant further investigation.
This paper is a departure from a 2012 paper by doctors at Tufts, which concluded that myocardial crypts were associated with HCM, and that they were often found in relatives of HCM patients found to be gene positive for HCM, but lacking the hallmark thickening of the ventricle.
Here is an example of what the crypts look like on MRI.
If you are looking for a good survey of current practices in the treatment of HCM, a recent article published in the journal Structural Heart by Dr. Ahmad Masri and the team at Oregon Health and Sciences University (OHSU) provides an informative overview of thirty controversies and considerations in the treatment of HCM. This article explains in some detail how the doctors at this HCM Center approach these situations.
Continue reading “HCM Treatment: The View from OHSU”