https://doi.org/10.1007/s00392-025-02625-4
1Universitätsklinikum Essen Klinik für Kardiologie und Angiologie Essen, Deutschland; 2Universitätsklinik Essen Klinik für Hämatologie und Stammzelltransplantation Essen, Deutschland
Introduction:
Cardiac amyloidosis, including both wild-type and hereditary forms, is a progressive infiltrative cardiomyopathy characterized by amyloid deposits in the myocardium. Current treatments are limited, with transthyretin stabilizers as the only specific therapies available, while conventional heart failure treatments have shown minimal efficacy. This study evaluates the tolerability and potential benefits of mineralocorticoid receptor antagonists (MRAs) in patients with transthyretin cardiomyopathy (ATTR-CM).
Methods:
Consecutive ATTR-CM patients receiving transthyretin stabilizer therapy were analyzed at baseline, 6 months, and 12 months. Data included normally distributed variables reported as mean ± standard deviation and non-normally distributed data as median (quartile 1-3). Statistical analyses involved two-sided t-tests, Mann-Whitney U tests, and Chi-square tests, with p < 0.05 deemed significant. Ethics approval was granted by the University of Duisburg-Essen (23-11500-BO).
Results:
Screening identified 194 patients diagnosed with ATTR-CM, with 75 (39%) patients received MRA therapy. Mean age was 79 years, with 173 (87%) male patients, without differences between MRA and non-MRA groups. Coronary heart disease affected approximately half of both groups, with no difference in prevalence (p = 0.973).
Left ventricular ejection fraction (LVEF) was similar, averaging 50% in the MRA group versus 52.8% in the non-MRA group (p = 0.641). Baseline levels of creatinine and NTproBNP were comparable across groups, with no significant changes observed at 6 (both p > 0.999) or 12 months (creatinine p = 0.298, NTproBNP p > 0.999). Echocardiographic parameters, including stroke volume, remained stable in the MRA group (p > 0.999). Patients on MRAs showed a non-significant trend towards improvement in NYHA functional class over time. Initially, 52.1% were in NYHA Class I–II, increasing to 60% by 6 months and 63% by 12 months, though these changes were not statistically significant (p > 0.05 at all time points). Notably, patients receiving MRAs did not experience hypotension during follow-up (mean blood pressure: 98.7 mmHg ± 15.8 at 6 months (p = 0.999); 99.1 mmHg ± 13.1 at 12 months (p > 0.999)), and therapy discontinuation due to low blood pressure was not necessary. Discontinuation of transthyretin stabiliser therapy rate was also very low after 12 months (MRA group: n = 3 (4%), Non-MRA group: n = 10 (8%) (p = 0.232)).
Conclusion:
MRAs appear to be a well-tolerated treatment option for ATTR-CM patients, with no observed increase in renal function deterioration or hypotension. Prospective studies are recommended to further explore the efficacy and safety of MRAs in this patient population, including trials with newer, non-steroidal MRA substances.