New findings from a large, international registry suggest that in patients with the dual pathology of aortic stenosis and transthyretin-associated cardiac amyloidosis, a combination of aortic valve replacement and disease-specific medication offers the most significant survival benefit.¹ The study (NCT06129331) aimed to clarify the prognostic impact of these treatments in this increasingly recognised patient population.
This multicentre, international registry included 226 patients from 16 centres with a concomitant diagnosis of significant aortic stenosis (AS) and transthyretin-associated cardiac amyloidosis (ATTR-CA). AS was diagnosed via transthoracic echocardiography, and ATTR-CA was confirmed by myocardial uptake on bone scintigraphy or endomyocardial biopsy.
The cohort had a mean age of 85 years, and 80.4% were male. Valve treatment strategies included transcatheter or surgical aortic valve replacement (AVR) in 75.2% of patients, while 23.5% were managed conservatively. ATTR-specific medication, predominantly tafamidis, was administered to 30.5% of patients. The primary clinical endpoints were all-cause mortality, cardiovascular (CV) mortality, and hospitalisation for heart failure (HHF).
Over a mean follow-up period of 3.6 years, 49.6% of patients died, with 79.5% of deaths being from CV causes.
The administration of ATTR-specific medication was independently associated with a significant reduction in both all-cause mortality (weighted hazard ratio [HR] 0.40, 95% confidence interval [CI] 0.24–0.68) and CV mortality (weighted HR 0.47, 95% CI 0.27–0.83). However, it was not associated with a reduction in HHF.
Similarly, AVR was associated with improved survival in the overall cohort (HR 0.60, 95% CI 0.39–0.93) and specifically in the severe AS cohort (HR 0.42, 95% CI 0.26–0.70).
The most favourable prognosis was observed in patients who received both ATTR-specific medication and AVR. The survival rate in this group was comparable to that of a matched control cohort of patients with lone AS who underwent AVR.
These findings suggest that both AVR and ATTR-specific therapy provide a significant survival benefit for patients with dual AS and ATTR-CA pathology. The data support a dual-treatment approach to manage both conditions concurrently, potentially normalising mid-term outcomes to the level of treated lone AS. The results also highlight the clinical importance of actively screening for ATTR-CA in patients presenting with AS to ensure they receive optimal, life-prolonging therapies.
This study was funded by Pfizer.
References
1. Nitsche C, Dobner S, Rosenblum HR, et al. Cardiac transthyretin amyloidosis treatment improves outcomes after aortic valve replacement for severe stenosis. Eur Heart J 2025;46(44):4795–4806. https://doi.org/10.1093/eurheartj/ehaf362
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