What the trial was testing
The VALOR-HCM enrolled 112 patients with hypertrophic cardiomyopathy. The study was sponsored by Bristol-Myers Squibb and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
93% of patients no longer needed surgery after long-term mavacamten.
JAMA Cardiology · 2023 · NCT04349072
These findings — that for septal reduction at 56 weeks on long-term mavacamten in severe HCM — were published in the JAMA Cardiology and represent the headline result of the study.
Researchers tracked outcomes across 112 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with hypertrophic cardiomyopathy, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Mavacamten (Camzyos) is FDA-approved and available now and can reduce the need for septal reduction surgery in severely symptomatic obstructive hypertrophic cardiomyopathy. Regular echocardiograms are required because it can lower heart pumping in some patients. Ask a cardiologist familiar with HCM about access.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open hypertrophic cardiomyopathy trials
DZHK TORCH-Plus is a Registry for Patients With Cardiomyopathies and Serves as Source for Cardiovascular Research Studies
The DZHK TranslatiOnal Registry for CardiomyopatHies (DZHK TORCH) represents a unique resource of clinical data and high quality biological samples to enable innovative clinical and molecular studies on cardiomyopathies (CMP). As a multi-center German cardiomyopathy registry, TORCH has been prospectively admitting patients since December 2014. 2,300 patients were recruited as planned. Taken together, patient data showed that the prevalence of these diseases is much higher in men than in women, atrial fibrillation is common in all forms of CMPs as well as rare forms of disease indicate a higher risk and higher morbidity. This DZHK TORCH register is now to be expanded with a second phase (DZHK TORCH-Plus). The second phase DZHK TORCH-Plus consists of 4 main modules: 1. "Clinical phenotyping, follow-up \& biosampling" 2. "Genomics", 3. "Inflammation" and 4. "Biomarker". The central aims are 1) to significantly increase the number of probands (n = 4340) in order to better address the different types of CMPs, especially patients with rare CMP forms such as LVNC and ARVC or with probably molecularly explainable cardiomyopathies (familial DCM), 2) to prolong the longitudinal with a further follow-up to achieve sufficient events and thereby derive clinical recommendations for risk assessment, 3) to increase the number of probands with state-of-the-art phenotyping, 4) to pinpoint the effect of myocardial inflammation, fibrosis, gender and to determine or predict genotypes based for outcome, 5) to validate novel biomarkers developed in other DZHK studies, and 6) to foster active cooperation with international CMP registries and partners from industry.
Real-world Patient Reported Outcomes Among Patients Treated With Camzyos
This real-world study will assess changes in health status among participants with symptomatic obstructive hypertrophic cardiomyopathy who are treated with mavacamten in the real world.