Cardiac Biomarker Change at 1 Year After Tafamidis Treatment and Clinical Outcomes in Patients With Transthyretin Amyloid Cardiomyopathy

J Am Heart Assoc. 2024 May 21;13(10):e034518. doi: 10.1161/JAHA.124.034518. Epub 2024 May 18.


Background: Although tafamidis treatment improves prognosis in patients with wild-type transthyretin amyloid cardiomyopathy, an optimal surrogate marker monitoring its therapeutic effect remains unclear. This study investigated the association between changes in cardiac biomarkers, high-sensitivity cardiac troponin T (hs-cTnT) and B-type natriuretic peptide (BNP) during the first year after tafamidis treatment and clinical outcomes.

Methods and results: In 101 patients with wild-type transthyretin amyloid cardiomyopathy receiving tafamidis at our institution, change in cardiac biomarkers from baseline to 1 year after tafamidis administration and its association with composite outcomes (composite of all-cause death and hospitalization attributable to heart failure) was assessed. During the follow-up period (median, 17 months), 16 (16%) patients experienced composite outcomes. The hs-cTnT level significantly decreased at 1 year after tafamidis treatment, unlike the BNP level. The frequencies of increased hs-cTnT and BNP levels were significantly higher in those with composite outcomes than in those without (44% versus 15%; P=0.01). Kaplan-Meier survival analysis showed that patients in whom both hs-cTnT and BNP levels increased at 1 year after tafamidis had a higher probability of composite outcomes compared with those with decreased hs-cTnT and BNP levels (log-rank P<0.01). Cox regression analysis identified increased hs-cTnT and BNP levels at 1 year after tafamidis administration as an independent predictor of higher cumulative risk of composite outcomes.

Conclusions: Deterioration in cardiac biomarkers during the first year after tafamidis treatment predicted a worse prognosis, suggesting the utility of serial assessment of cardiac biomarkers for monitoring the therapeutic response to tafamidis in patients with wild-type transthyretin amyloid cardiomyopathy.

Keywords: B‐type natriuretic peptide; high‐sensitivity cardiac troponin T; tafamidis; wild‐type transthyretin amyloid cardiomyopathy.

MeSH terms

  • Aged
  • Aged, 80 and over
  • Amyloid Neuropathies, Familial* / blood
  • Amyloid Neuropathies, Familial* / diagnosis
  • Amyloid Neuropathies, Familial* / drug therapy
  • Amyloid Neuropathies, Familial* / mortality
  • Benzoxazoles* / therapeutic use
  • Biomarkers* / blood
  • Cardiomyopathies* / blood
  • Cardiomyopathies* / diagnosis
  • Cardiomyopathies* / drug therapy
  • Cardiomyopathies* / mortality
  • Female
  • Heart Failure / blood
  • Heart Failure / drug therapy
  • Heart Failure / mortality
  • Humans
  • Male
  • Middle Aged
  • Natriuretic Peptide, Brain* / blood
  • Prealbumin / metabolism
  • Retrospective Studies
  • Time Factors
  • Treatment Outcome
  • Troponin T* / blood


  • tafamidis

Supplementary concepts

  • Amyloidosis, Hereditary, Transthyretin-Related