Heart failure & cardiomyopathies

Clinical Characteristics, Outcome, and Therapeutic Effect of Tafamidis in Wild-Type Transthyretin Amyloid Cardiomyopathy

ESC Heart FailPublished Retrospective cohort study

Main findingResult — go to the resultHazard ratio 0.31 95% CI 0.11–0.93

Authors' conclusion

The prognosis of patients with ATTRwt-CM treated with tafamidis was more favourable than that of untreated patients.

Study context

Population
Consecutive patients with wild-type transthyretin amyloid cardiomyopathy (ATTRwt-CM)
Participants
125 vs 55
Exposure
Tafamidis vs no treatment
  • Treatmentn = 125
  • Treatment-naïven = 55reference
Outcome
All-cause mortality and HF hospitalization
Methodological detailssingle-centre, propensity-score matched, inverse probability of treatment weighting
  • Single-centre
  • Propensity-score matched
  • Inverse probability of treatment weighting

Main finding

As reported in the abstract

Hazard ratio

0.31

95% CI 0.11–0.93

P = 0.04

Treatmentvs Treatment-naïve
HR 0.31 (95% CI 0.11–0.93); P = 0.04
More favourable all-cause mortality and HF hospitalization with treatment, entire cohort
P < 0.01
More favourable all-cause mortality and HF hospitalization with treatment, propensity score-matched cohort
P < 0.05
Reduced all-cause mortality (Kaplan-Meier, log-rank), curves diverging after ≈18 months, matched cohort
P = 0.03

Observational data: associations, not effects.

Further findings

High risk score (2-3 points: High-sensitivity cardiac troponin T (hs-cTnT) >0.05 ng/mL, BNP >250 pg/mL, estimated glomerular filtration rate (eGFR) <45 mL/min/1.73 m²) and composite of all-cause death and HF hospitalization, treatment group

HR (95% CI)P value
High score (2-3 points)Multivariate logistic regression1.55 (1.22–1.98)P < 0.01
OutcomeBaseline12 months
hs-cTnT, treatment group · 12 months0.054 (range or interval as printed 0.036–0.082)0.044 (range or interval as printed 0.033–0.076)
hs-cTnT, treatment group — hs-cTnT decreased significantly
P = 0.002

BNP, echocardiographic parameters, native T1, extracellular volume fraction on CMR · 12 months

No significant changes

Original publication

Clinical Characteristics, Outcome, and Therapeutic Effect of Tafamidis in Wild-Type Transthyretin Amyloid Cardiomyopathy. ESC Heart Fail. Published 18 April 2023. doi:10.1002/ehf2.14380

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