Related Experiment Video

Updated: Jan 13, 2026

Comprehensive Echocardiographic Assessment of Right Ventricle Function in a Rat Model of Pulmonary Arterial Hypertension
07:38

Comprehensive Echocardiographic Assessment of Right Ventricle Function in a Rat Model of Pulmonary Arterial Hypertension

Published on: January 20, 2023

4.5K

Baseline clinical profile of patients with obstructive hypertrophic cardiomyopathy in the Italian Mavacamten early

G Bonacchi1, A Del Franco1, P G Agostoni2

  • 1Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy.

International Journal of Cardiology
|January 10, 2026
PubMed

Insights

Mavacamten, a novel therapy for obstructive hypertrophic cardiomyopathy (oHCM), shows promise in a compassionate use program. This treatment offers an option for patients with advanced disease, particularly those ineligible for surgery.

Area of Science:

  • Cardiology
  • Pharmacology
  • Genetics

Background:

  • Obstructive hypertrophic cardiomyopathy (oHCM) is a significant cardiovascular condition.
  • Mavacamten represents a novel, disease-specific therapy targeting oHCM.
  • Conventional treatments often leave patients symptomatic, highlighting the need for new therapeutic strategies.

Purpose of the Study:

  • To evaluate the early engagement and characteristics of patients receiving mavacamten through an Early Access Program (EAP) in Italy.
  • To assess the clinical and echocardiographic profile of oHCM patients treated with mavacamten.
  • To determine the proportion of patients eligible for septal reduction therapy (SRT) within the EAP cohort.

Main Methods:

  • Prospective collection of clinical and echocardiographic data from 337 oHCM patients in Italy.
  • Comparison of the EAP cohort characteristics with the EXPLORER-HCM trial cohort.
  • Analysis of patient eligibility for SRT and associated echocardiographic parameters.

Main Results:

  • The Italian EAP cohort (337 patients) was older and presented with a more advanced cardiac phenotype than the EXPLORER-HCM cohort.
  • Lower left ventricular ejection fraction (LVEF) and higher left atrial volume index (LAVI) were observed in the EAP group.
  • Only 22% of patients were candidates for SRT, with higher LVOT obstruction gradients in this subgroup.

Conclusions:

  • The mavacamten EAP in Italy demonstrated substantial patient engagement.
  • The Italian oHCM cohort exhibited more advanced disease characteristics compared to a previous trial.
  • Mavacamten offers a valuable therapeutic option for symptomatic oHCM patients, especially those unsuitable for SRT.
Abstract

Keywords:
Baseline characterizationEarly access programHCMMavacamten

More Related Videos

Investigating the Pathogenesis of MYH7 Mutation Gly823Glu in Familial Hypertrophic Cardiomyopathy using a Mouse Model03:45

Investigating the Pathogenesis of MYH7 Mutation Gly823Glu in Familial Hypertrophic Cardiomyopathy using a Mouse Model

Published on: August 8, 2022

4.2K
Isolation and Characterization of Cardiac Mesenchymal Stromal Cells from Endomyocardial Bioptic Samples of Arrhythmogenic Cardiomyopathy Patients09:16

Isolation and Characterization of Cardiac Mesenchymal Stromal Cells from Endomyocardial Bioptic Samples of Arrhythmogenic Cardiomyopathy Patients

Published on: February 28, 2018

8.0K

Related Concept Videos

Cardiomyopathy III: Hypertrophic Cardiomyopathy01:29

Cardiomyopathy III: Hypertrophic Cardiomyopathy

Hypertrophic cardiomyopathy, or HCM, is an autosomal dominant genetic disorder characterized by asymmetric left ventricular hypertrophy without ventricular dilation. It is more common in men and is typically diagnosed in young, athletic adults.EtiologyHCM is primarily genetic and is caused by mutations in genes encoding sarcomeric proteins. Researchers have identified over 1400 mutations across at least 11 different genes. Among these, the most frequently occurring mutations are found in the...
387
Acute Coronary Syndrome III: Diagnostic Studies01:30

Acute Coronary Syndrome III: Diagnostic Studies

Diagnosing acute coronary syndrome or ACS begins with a thorough patient history. Notable symptoms include central, crushing chest pain radiating to the left arm, neck, jaw, or back, along with shortness of breath, sweating (diaphoresis), nausea, vomiting, dizziness, and palpitations.It is crucial to note any history of cardiac illnesses and assess risk factors, including age, gender, smoking, hypertension, diabetes, hyperlipidemia, and a sedentary lifestyle.During physical examination, vital...
204
Cardiomyopathy IV: Restrictive Cardiomyopathy01:29

Cardiomyopathy IV: Restrictive Cardiomyopathy

Restrictive cardiomyopathy (RCM) is a rare heart muscle disease characterized by impaired ventricular filling due to stiffened ventricular walls, leading to significant diastolic dysfunction.EtiologyRestrictive cardiomyopathy can arise from both inherited and acquired diseases, many of which are systemic. It is categorized into four main types: infiltrative, storage, non-infiltrative, and endomyocardial diseases.Infiltrative diseases, such as amyloidosis, lead to RCM by depositing amyloid...
439
Cardiomyopathy II: Dilated Cardiomyopathy01:30

Cardiomyopathy II: Dilated Cardiomyopathy

Dilated cardiomyopathy, or DCM, is a progressive myocardial disorder characterized by ventricular chamber dilation and contractile dysfunction.EtiologyVarious factors can cause DCM, including hypertension and heavy alcohol intake, which contribute to the weakening and enlargement of the heart muscle. Viral infections, such as Coxsackievirus B, adenoviruses, and influenza, can lead to DCM by causing inflammation and damage to heart tissue. Certain chemotherapeutic agents, including daunorubicin,...
455
Mitral Stenosis II: Clinical features and Diagnostic Tests01:23

Mitral Stenosis II: Clinical features and Diagnostic Tests

Mitral stenosis is a heart condition in which the mitral valve, which allows blood to flow from the left atrium to the left ventricle, becomes narrowed or stenotic. This narrowing hinders blood flow and leads to clinical symptoms requiring specific medical evaluations and management strategies. The following overview outlines the clinical symptoms, assessments, diagnostic findings, prevention methods, and treatments for mitral stenosis.Clinical ManifestationsDyspnea (shortness of breath): This...
209
Cardiomyopathy I: Introduction and Classification01:25

Cardiomyopathy I: Introduction and Classification

Cardiomyopathy, or CMP, is a group of diseases affecting the myocardial structure, impairing its ability to pump blood effectively. This condition can lead to arrhythmias, heart failure, or sudden cardiac death.Cardiomyopathies are classified into primary and secondary categories:Primary Cardiomyopathy refers to conditions involving only the heart muscle that are often idiopathic (of unknown cause) or genetic. They primarily affect the myocardium without the involvement of other systemic...
490