Valsartan, an angiotensin II receptor inhibitor, delayed disease progression and improved heart structure and function in patients with early-stage hypertrophic cardiomyopathy, according to results of a recent published clinical trial in Natural medicine.
This is the first time that we have identified a treatment that can actually have an impact on the underlying disease process, as opposed to just treating the symptoms. Moreover, if we can delay the progression of the disease, it may ultimately mean that we can have a significant impact on the risk of sudden cardiac death in this population. “
Philip Thrush, MD, ’13 GME, associate professor of pediatrics, division of cardiology and study co-author
Hypertrophic cardiomyopathy is characterized by abnormal thickening of the heart muscle. The disease results from mutations in the genes that code for sarcomeres, protein filaments essential for the proper contraction of the heart muscle.
While most patients are asymptomatic, the disease is characterized by left ventricular hypertrophy (when the left ventricle of the heart is unable to pump blood properly), myocardial fibrosis (scarring in the heart muscle), and an increased risk of heart failure and arrhythmias.
Current treatments for the disease only treat its symptoms and aim to slow or regulate the patient’s heart rate. Therefore, therapies that slow the progression of the disease and address its underlying causes are badly needed, and the drug valsatran, an angiotensin II receptor blocker, has been suggested as a solution to completely prevent the disease. development of hypertrophy.
Previous work had shown that a drug similar to angiotensin II receptors prevented the development of hypertrophy when given to mouse models with early stage disease, suggesting that valsartan could also be effective treatment.
In the current clinical trial, over 170 participants aged 8 to 45 who were diagnosed with early stage hypertrophic cardiomyopathy were randomized to receive either valsartan or placebo for two years. Participants were followed at one and two years for changes in certain cardiovascular characteristics, including the thickness, mass and volume of the left ventricular wall.
Overall, participants in the valsartan group demonstrated improved cardiac structure and function compared to the placebo group. The drug was also well tolerated by the participants, according to the authors, encouraging further investigation into the long-term effects of the drug and determining the optimal time to administer the drug to patients.
“While we know it’s beneficial to start early, we don’t know how beneficial it is,” Thrush said. “We have many children and young adults who are known to carry disease-causing familial mutations but continue to have normal echocardiograms, ECGs and even cardiac MRI scans, so it can be difficult to generalize these findings to a population. completely asymptomatic. It is certainly a challenge. with such a rare disease, but as a field we owe it to these patients to push the boundaries of medicine. “
Ho, CY, et al. (2021) Valsartan in early-stage hypertrophic cardiomyopathy: a randomized phase 2 trial. Natural medicine. doi.org/10.1038/s41591-021-01505-4.