Inclusion and Exclusion Criteria3

Study Design   >   Inclusion and Exclusion Criteria

Inclusion Criteria

  • Patients between 18 and 90 years old with ATTRwt or hATTR
  • ATTR-CM confirmed by biopsy from cardiac and non-cardiac sites
  • ATTRwt confirmed on IHC, scintigraphy, or MS
  • Cardiac involvement confirmed on ECHO with end-diastolic IVS wall thickness >12mm
  • History of heart failure (HF), ≥1 hospitalization for HF or clinical evidence of HF (signs or symptoms of volume overload or elevated intra-cardiac pressures requiring treatment with a diuretic for improvement)
  • NT-proBNP level ≥600 pg/mL
  • 6-minute walk-test distance >100 meters

Exclusion Criteria

  • HF that was not due to ATTR-CM
  • NYHA class IV heart failure
  • Presence of light-chain amyloidosis
  • History of liver or heart transplantation
  • Implanted mechanical cardiac assist device
  • Previous treatment with tafamidis
  • eGFR < 25 mL/min/1.73 m2
  • Liver transaminase levels >2 x ULN
  • Severe malnutrition as defined by a mBMI <600 kg/m2·g/L
  • Concurrent treatment with non-steroidal anti-inflammatory drugs, tauroursodeoxycholate, doxycycline, calcium channel blockers, or digitalis

ATTR-CM: Transthyretin amyloid cardiomyopathy; ATTRwt: wild-type ATTR-CM; ECHO: echocardiography; eGFR: estimated glomerular filtration rate; hATTR: hereditary ATTR-CM;
IHC: immunohistochemistry; IVS: interventricular septal; mBMI: modified body-mass index; MS: mass spectrometry; NYHA: New York Heart Association;
NT-proBNP: N-terminal pro-B-type natriuretic peptide; ULN: upper limit of the normal range; TTR: transthyretins