Abstract 12239: Improvement in Measures of Disease Progression With Tafamidis in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT)
Bibliographic record
Abstract
Introduction: Patients with transthyretin amyloid cardiomyopathy (ATTR-CM) experience a progressive decline in health and quality of life. Tafamidis is an effective treatment for patients with ATTR-CM, and was shown to reduce mortality and CV-related hospitalization in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT). As ATTR-CM is a progressive disease, either stabilization or improvement signifies an efficacious treatment. Methods: This was a post-hoc analysis of the proportion of patients in ATTR-ACT who stabilized or improved in clinical parameters. In ATTR-ACT, 441 adult patients with ATTR-CM (variant or wild-type) were randomized (2:1:2) to tafamidis 80 mg, tafamidis 20 mg, or placebo for 30 months, with pooled tafamidis (n=264) compared with placebo (n=177). Change from baseline was assessed at each study visit to determine patients who improved/did not change in: 6MWT distance; KCCQ-OS score; NT-proBNP concentration; PGA scale; and NYHA class. Any patient with missing data was considered not improved. Results: A significantly greater proportion of patients improved/did not change with tafamidis compared with placebo for each outcome at Month 30 ( Table ). Of patients who started treatment with tafamidis, 20-27% improved in 6MWT distance, KCCQ-OS score, and NT-proBNP concentration at Month 30, compared with 5-10% of patients with placebo. There were greater proportions who improved in these measures at earlier timepoints (32-42% with tafamidis vs 19-24% with placebo at Month 12; P <0.01 for all). Conclusions: While patients with ATTR-CM are expected to experience a progressive decline over time, a significant proportion of tafamidis-treated patients in ATTR-ACT had improvements in measures of functional capacity, health-related quality of life, and cardiac biomarkers within 12 months and through the study. These data provide further evidence of the clinical benefits with tafamidis in patients with ATTR-CM.
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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 | 0.002 | 0.001 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.001 | 0.001 |
| Insufficient payload (model declined to judge) | 0.007 | 0.001 |
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".