Table 2.
Clinical Outcomes With Tafamidis in ATTR-CM: ATTR-ACT vs. Real-World Evidence
| Outcome | ATTR-ACT6 | Real-world studies7–9,22–32 | Summary |
|---|---|---|---|
| All-cause mortality | Reduced by ~30% vs. placebo at 30 months |
Reduced mortality, especially with early treatment |
Benefit seen in both settings, more with early initiation |
| Cardiovascular hospitalizations | Fewer hospitalizations vs. placebo |
Generally reduced; evident after ≥12 months of therapy |
Hospitalization reduction confirmed in several cohorts |
| Functional capacity (6MWT, V̇O2) |
Slower decline | Stabilized; improvement in early or lower-functioning patients |
6MWT and CPET support functional preservation |
| Quality of life (KCCQ-OS) | Maintained | Improvement or stabilization reported |
Reflects patient-perceived benefit |
| NT-proBNP/troponin | Slower biomarker progression |
Stabilized or declined in early treated patients |
Biomarker trends reflect clinical stabilization |
| Echocardiography/CMR imaging |
Modest structural change | Stable or reduced LV mass/strain decline in several cohorts |
Dependent on disease stage at treatment start |
| Adverse events | Similar to placebo | Well tolerated across real-world cohorts |
Favorable safety profile in routine care |
6MWT, 6-min walk test; ATTR-CM, transthyretin amyloid cardiomyopathy; CMR, cardiac magnetic resonance; CPET, cardiopulmonary exercise testing; KCCQ, Kansas City Cardiomyopathy Questionnaire; LV, left ventricle; NT-proBNP, N-terminal pro B-type natriuretic peptide; V̇O2, oxygen uptake.