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
Taiwan Registry of Hypertrophic Cardiomyopathy (THIC) Research Synopsis
Hypertrophic cardiomyopathy (HCM) is hallmarked by the presence of left ventricular hypertrophy (LVH) and may present various symptoms including arrhythmia and heart failure. Mutations in the genes related to sarcomeric proteins and metabolic disorders are known causes of HCM. However, it remains required to further explore the prevalence of HCM in the context of Taiwanese' genetic background. Additionally, certain rare diseases that affect the heart, including Fabry disease, cardiac amyloidosis, may present LVH, which makes precise diagnosis among HCM and these diseases more challenging. In this TSOC multi-center registry, we aim to systematically evaluate the clinical, genetic, biochemical features,prevalence, and possible natural course of HCM and relevant rare diseases such as Fabry disease in Taiwan. In the meanwhile, we may also generate the specific "red-flag" signs of Fabry disease in Taiwan.
Mavacamten Post-marketing Surveillance in Patients With Obstructive Hypertrophic Cardiomyopathy in Japan
The purpose of this study is to assess the real-world effectiveness and safety of mavacamten in patients with obstructive hypertrophic cardiomyopathy (HCM) receiving mavacamten in Japan