Myosin-ATPase inhibitor in real-world patients with obstructive HCM: a report by the Cardiomyopathies and Pericardial Diseases WG of the Italian Society of Cardiology
Bibliographic record
Abstract
INTRODUCTION: Approximately two-thirds of patients suffering from hypertrophic cardiomyopathy present with an obstructive (HOCM) physiology. For years, medical therapy has been limited to beta blockers, verapamil and/or disopyramide. Recently, a novel class of drugs, the allosteric inhibitors of the cardiac-specific myosin head adenosine triphosphatase (ATPase), have been demonstrated to be effective in relieving the dynamic obstruction and related clinical condition. In July 2024 the Cardiomyopathies and Pericardial Diseases WG of the Italian Society of Cardiology started with a nationwide multicentre registry aimed at investigating the pathophysiology of dynamic obstruction in real-world patients with HOCM. Based on the medical records, this brief report deals with the proportion of patients who were eligible for Mavacamten based on the Explorer-HCM entry criteria, and then admitted for compassionate use by the end of 2024. METHODS AND RESULTS: The Hypertrophic Obstructive Physiology Study (HOPS) was designed as a registry on consecutive adult patients admitted to 19 tertiary Cardiac Centres in Italy until June 2024. A total of 424 patients, 53% males, aged 64 ± 13 years, were included. We retrospectively recognized 200 Mavacamten-eligible patients (47.2%) on 5 Explorer-HCM requirements. Forty out of this latter group, along with 15 more patients on 4 criteria, were admitted to the compassionate use programme ( n = 55, 13% of the whole population). Forty-three showed subaortic obstruction and 12 a mid-ventricular variant. Ethical committee approval items varied among centres and regions. DISCUSSION: This study confirmed our recent demonstration that approximately half of real-world HOCM patients are suitable for Mavacamten therapy based on the full Explorer-HCM trial entry criteria. Due to the current limitation of compassionate use programmes in Italy, only one in four patients was admitted for treatment.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.002 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.000 | 0.002 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one teacher head, not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".