Abstract 4360554: Mavacamten monotherapy in real-world patients with obstructive hypertrophic cardiomyopathy: Evidence from COLLIGO-HCM
Bibliographic record
Abstract
Background: The mavaCamten ObservationaL evIdence Global cOnsortium in hypertrophic cardiomyopathy (COLLIGO-HCM, ClinicalTrials.gov ID NCT06372457) is a global observational study describing real-world outcomes of obstructive HCM patients treated with mavacamten. Limited real-world evidence exists regarding the use of mavacamten as monotherapy or following reduction or withdrawal of standard of care background therapies such as beta-blockers or calcium channel blockers. Purpose: To describe the real-world outcomes of patients treated with mavacamten monotherapy. Methods: This retrospective study uses patient-level data from existing medical records and electronic registries from COLLIGO-HCM sites in the US, Canada, the UK, Australia, and Israel. Patient characteristics, NYHA class, echocardiography data, and safety were analyzed at baseline and follow-up visits, up to week 36 in four cohorts: monotherapy group (n=88), defined as those with monotherapy at initiation (n=20) plus monotherapy after discontinuation of background therapy (n=68), mavacamten in combination with background therapy that has been reduced (n=13), mavacamten in combination with stable, unchanged background therapy over the duration of the study period (n=177), and the overall cohort (n= 278). Results: The mavacamten monotherapy cohort included 88/278 patients (31.7%). Baseline clinical and demographic characteristics include: 55.7% female, 56.8% NYHA Class II and 43.2% Class III, high BMI (mean: 31.6 kg/m 2 ), history of atrial fibrillation (15.9%) and hypertension (71.6%). The median follow-up time was 35.9 weeks (IQR:21.9,116.9) (Table 1). By week 36, 60% of patients achieved ≥ 1 NYHA class improvement. Additionally, mean left ventricular outflow tract (LVOT) gradients of ≤30 mm Hg at rest and with Valsalva were achieved in 97.3% and 81.6% of patients, respectively (Table 2. Figure). Mean left ventricular ejection fraction (LVEF) post mavacamten initiation remained at or above 62% throughout follow-up (baseline value 66.4%). Two patients in the monotherapy cohort permanently discontinued mavacamten due to LVEF<50%, both recovered after discontinuing treatment. Conclusions: This analysis demonstrates effectiveness, safety and consistency of mavacamten monotherapy with previously reported monotherapy analyses from pivotal, long-term and real-world studies.
Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.
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 | 0.001 |
| 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) | 0.002 | 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".