Mavacamten Monotherapy in Real-World Patients With Obstructive Hypertrophic Cardiomyopathy: Evidence From COLLIGO-HCM
Bibliographic record
Abstract
BACKGROUND: Mavacamten has been shown to improve cardiac function and symptoms in patients with symptomatic (New York Heart Association class II-III) obstructive hypertrophic cardiomyopathy (HCM). Clinical studies suggest that mavacamten monotherapy is efficacious and has a favorable safety profile, but limited evidence exists regarding monotherapy in real-world studies. This analysis aimed to describe the effectiveness and safety outcomes of mavacamten monotherapy in the real-world COLLIGO-HCM study (Mavacamten ObservationaL Evidence Global Consortium in Hypertrophic Cardiomyopathy). METHODS: Patient-level data recorded between April 2022 and February 2025 at 7 sites across 5 countries were extracted. Adult patients with a diagnosis of HCM from 2018 onwards were eligible for inclusion if they had ≥1 mavacamten prescription after the date of diagnosis. Patients were categorized based on background therapy status during mavacamten treatment: mavacamten monotherapy or mavacamten with background therapy (down-titration or no dose modification). RESULTS: Overall, 278 patients were included and received mavacamten (mavacamten monotherapy, n=88; mavacamten with background therapy, n=190). At month 9, most patients achieved ≥1 New York Heart Association class improvement from baseline (mavacamten monotherapy, 60.0%; mavacamten with background therapy, 61.0%). Improvements in resting and Valsalva left ventricular outflow tract gradients from baseline to month 9 were observed in both subgroups; mean left ventricular ejection fraction through month 9 remained ≥62.0% with mavacamten monotherapy and ≥61.4% with mavacamten with background therapy. Two patients in the mavacamten monotherapy subgroup and 1 patient in the mavacamten with background therapy subgroup permanently discontinued treatment owing to left ventricular ejection fraction <50%. CONCLUSIONS: Mavacamten monotherapy was associated with improvements in cardiac function and symptoms, and positive benefits to the risk profile over a 9-month follow-up period; this was consistent with improvements observed in patients treated with mavacamten with background therapy. REGISTRATION: URL: https://www.clinicaltrials.gov; Unique identifier: NCT06372457.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.005 | 0.013 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.001 | 0.002 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.001 | 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 source (direct Gemma or distilled Codex), 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".