Tafamidis delays myocardial amyloid progression in transthyretin amyloid cardiomyopathy patients: Study
Austria: Treatment with tafamidis delays myocardial amyloid progression in patients with transthyretin amyloid cardiomyopathy (ATTR-CM), a recent study has found. This results in functional, structural, and clinical benefits compared to the natural course. Findings of this study, published in the European Heart Journal - Cardiovascular Imaging, indicate that serial cardiac magnetic resonance (CMR) including measurement of extracellular volume (ECV) may be suitable for disease-specific therapy monitoring.
Previous studies have shown tafamidis to be associated with improved outcomes in ATTR-CM patients. However, whether tafamidis affects cardiac amyloid deposition and structural changes in the myocardium is not yet known. Diana Bonderman, Medical University of Vienna, Waehringer Guertel Vienna, Austria, and colleagues, therefore, aimed to determine disease-modifying effects on myocardial amyloid progression and to identify imaging parameters that could be applied for specific therapy monitoring.
For the study, ATTR-CM patients underwent serial CMR imaging using T1 mapping techniques to derive ECV.
The study revealed the following findings:
- Patients receiving tafamidis 61 mg (n = 35) or 20 mg (n = 15) once daily showed stable measurements at follow-up (FU) {61 mg: 9.0 months, 20 mg: 11.0 months} in left ventricular (LV) ejection fraction (LVEF; 61 mg: 47.6% vs. 47.5%, P = 0.935; 20 mg: 52.4% vs. 52.1%), LV mass index (LVMI; 61 mg: 110.2 vs. 106.2 g/m2, P = 0.304; 20 mg: 114.5 vs. 115.4 g/m2), and ECV (61 mg: 47.5% vs. 47.7%; 20 mg: 56.7% vs. 57.5%), whereas treatment-naïve ATTR-CM patients (n = 19) had clear signs of disease progression at the end of the observation period [12.0 months; LVEF: 53.3% vs. 45.7%; LVMI: 98.9 vs. 106.9 g/m2; ECV: 49.3% vs. 54.6%].
- The between-group comparison at FU revealed positive effects in tafamidis 61 mg-treated compared to treatment-naïve patients, while those treated with 20 mg showed no difference in the above LV measurements when compared with treatment-naïve.
- Both treatment groups showed clinically beneficial effects compared to the natural course [61 mg, 6-min walk distance (6-MWD), N-terminal prohormone of brain natriuretic peptide (NT-proBNP); 20 mg, 6-MWD, NT-proBNP].
"Tafamidis delays myocardial amyloid progression in ATTR-CM patients, resulting in structural, functional, and clinical benefits compared to the natural course," wrote the authors.
Reference:
René Rettl, Christopher Mann, Franz Duca, Theresa-Marie Dachs, Christina Binder, Luciana Camuz Ligios, Lore Schrutka, Daniel Dalos, Matthias Koschutnik, Carolina Donà, Andreas Kammerlander, Dietrich Beitzke, Christian Loewe, Silvia Charwat-Resl, Christian Hengstenberg, Johannes Kastner, Roza Badr Eslam, Diana Bonderman, Tafamidis treatment delays structural and functional changes of the left ventricle in patients with transthyretin amyloid cardiomyopathy, European Heart Journal - Cardiovascular Imaging, 2021;, jeab226, https://doi.org/10.1093/ehjci/jeab226
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