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BACKGROUND Mavacamten, a first-in-class cardiac myosin inhibitor, targets myosin adenosine triphosphatase to treat adults with moderate to severe symptomatic obstructive hypertrophic cardiomyopathy. Previous studies have demonstrated an approximate 50% reduction in left ventricular outflow tract pressure gradient in selected patients after 4 weeks of treatment. CASE REPORT We report the case of a 91-year-old man with hypertrophic cardiomyopathy, who presented with a longstanding history of progressive shortness of breath, unresponsive to treatment. After ruling out other potential causes such as coronary and pulmonary involvement, and performing serial ultrasound evaluations, we concluded that the worsening of his hypertrophic cardiomyopathy was the most likely underlying cause. After initiating Mavacamten therapy, the patient achieved a remarkable improvement, with over 80% reduction in left ventricular outflow tract obstruction observed within 4 weeks of treatment. He reports significant improvement in his shortness of breath both at rest and during daily activities. Dose titration and safety evaluation were performed through robust echocardiographic monitoring. CONCLUSIONS While our patient did not undergo genetic testing due to its high cost and limited clinical relevance, we believe that his remarkable response to this therapy may be linked to a genotype susceptibility enhancing the drug's pharmacodynamic effects, as suggested in the literature. This may have contributed to significant symptom relief and an improved quality of life. Further research is needed to better understand how genotype and phenotype influence treatment response, which could help refine our approach to optimizing non-invasive medical therapies.
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http://dx.doi.org/10.12659/AJCR.946956 | DOI Listing |
Biomed Environ Sci
August 2025
Department of Cardiology, Fuwai Central China Cardiovascular Hospital, Henan Provincial People's Hospital Heart Center, Zhengzhou 451464, Henan, China.
Hypertrophic cardiomyopathy (HCM) is a major contributor to cardiovascular diseases (CVD), the leading cause of death globally. HCM can precipitate heart failure (HF) by causing the cardiac tissue to weaken and stretch, thereby impairing its pumping efficiency. Moreover, HCM increases the risk of atrial fibrillation, which in turn elevates the likelihood of thrombus formation and stroke.
View Article and Find Full Text PDFJ Cardiovasc Pharmacol
September 2025
Graduate School of Cardiology, Bengbu Medical University, Bengbu 233000, Anhui, China.
Chronic stress-induced cardiac hypertrophy remains a critical precursor to heart failure, with current therapies limited by incomplete mechanistic targeting. Cyclin-dependent kinases (CDKs), pivotal regulators of cell cycle and stress signaling, are emerging therapeutic targets in cardiovascular pathologies. Using bioinformatics analysis of human hypertrophic cardiomyopathy datasets (GSE5500, GSE136308) and a murine transverse aortic constriction (TAC) model, we investigated the therapeutic effects of the CDK inhibitor R547 (10 mg/kg, intraperitoneal every 3 days) on pressure overload-induced cardiac remodeling.
View Article and Find Full Text PDFJTCVS Open
August 2025
Department of Cardiovascular Medicine, Mayo Clinic, Rochester, Minn.
Background: Proper risk stratification tools for patients with obstructive hypertrophic cardiomyopathy (oHCM) undergoing septal myectomy are lacking. Our objective was to assess the predictive value of preoperative N-terminal pro-brain natriuretic peptide (NT-proBNP) on perioperative outcomes and late survival in patients with oHCM undergoing transaortic septal myectomy.
Methods: Between 2008 and 2021, 834 patients with preoperative NT-proBNP measurements underwent septal myectomy.
Heart Rhythm
September 2025
Tufts Medicine CardioVascular Center, Division of Cardiology, Boston, MA.
Int J Cardiol
September 2025
Department of Demography and Geodemography, Faculty of Science, Charles University, Prague, Czech Republic.
Background: Alcohol septal ablation (ASA) is an established therapy for symptomatic hypertrophic obstructive cardiomyopathy (HOCM) in patients unresponsive to medical treatment. However, comprehensive assessment of ASA outcomes remains challenging. This study aimed to evaluate the impact of institutional experience and patient characteristics on achieving complete clinical and haemodynamic response (CCHR), a novel composite outcome integrating long-term symptomatic, haemodynamic, safety, and major clinical endpoints, including survival and resuscitation.
View Article and Find Full Text PDF