SPC365

Tafamidis to treat patients with transthyretin cardiac amyloidosis (ATTR-CM): real-world data from a tertiary centre

Session: Sessão de Posters 12 - Estratégias terapêuticas e desfechos em vida real na ATTR-CM
Speaker: Miguel Sobral Domingues

Congress: CPC 2026
Topic: F. Valvular, Myocardial, Pericardial, Pulmonary, Congenital Heart Disease
Theme: 17. Myocardial Disease
Subtheme: 17.4 Myocardial Disease – Treatment
Session Type: Posters Eletrónicos
FP Number: ---
Authors: Miguel Sobral Domingues; Rita Carvalho; Mariana Ramos; Sérgio Maltês; Tânia Laranjeira; Carlos Aguiar; Bruno M. Rocha

<p style="text-align:justify"><span style="font-size:12pt"><span style="font-family:Aptos,sans-serif"><strong><span style="font-family:&quot;Times New Roman&quot;,serif">Introduction:</span></strong></span></span></p>

<p style="text-align:justify"><span style="font-size:12pt"><span style="font-family:Aptos,sans-serif"><span style="font-family:&quot;Times New Roman&quot;,serif">Tafamidis 61 mg is the first therapy shown to improve survival and reduce cardiovascular hospitalizations in transthyretin cardiac amyloidosis (ATTR-CM). As real-world evidence continues to grow, further characterization of tafamidis use across diverse clinical settings remains important. Accordingly, we evaluated treatment patterns and outcomes in patients managed within a dedicated tertiary-care cardiomyopathy program.</span></span></span></p>

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<p style="text-align:justify"><span style="font-size:12pt"><span style="font-family:Aptos,sans-serif"><strong><span style="font-family:&quot;Times New Roman&quot;,serif">Methods:</span></strong></span></span></p>

<p style="text-align:justify"><span style="font-size:12pt"><span style="font-family:Aptos,sans-serif"><span style="font-family:&quot;Times New Roman&quot;,serif">We conducted a prospective, single-centre, all-comers study of consecutive patients with symptomatic Heart Failure (HF) due to ATTR-CM, up to November 2025. The diagnosis was performed based on the ESC multi-step algorithm. Management included HF therapy tailored to ATTR-CM (CHAD-STOP) and tafamidis 61 mg for patients who met the local protocol key criteria, including NYHA class I-II and no exclusion criteria [e.g., estimated glomerular filtration rate (GFR) &lt;25 mL/min and/or clinical frailty scale &gt;6]. The primary endpoint was a composite of cardiovascular hospitalization or all-cause death at 3 years. Subgroup analyses were performed according to the National Amyloidosis Centre (NAC) staging system. </span></span></span></p>

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<p style="text-align:justify"><span style="font-size:12pt"><span style="font-family:Aptos,sans-serif"><strong><span style="font-family:&quot;Times New Roman&quot;,serif">Results:</span></strong></span></span></p>

<p style="text-align:justify"><span style="font-size:12pt"><span style="font-family:Aptos,sans-serif"><span style="font-family:&quot;Times New Roman&quot;,serif">A total of 278 patients with ATTR-CM were included (median age 83 [78-87] years; 82% male; 8% hereditary ATTR-CM). Tafamidis 61mg was initiated in 173 patients (62.2%). Compared with patients on stand-alone HF treatment, those treated with tafamidis were significantly younger (median age: 82 [77-86]&nbsp; vs. 85 [81-89] years; p &lt;0.001) with better functional status (NYHA I-II: 93% vs. 50%; p&lt;0.001) and health status (e.g., Frailty Index &gt;3: 14% vs. 68%; p&lt;0.001), and having fewer comorbidities (e.g., median GFR 37 [31-50] vs. 31 [24-43] mL/min; p = 0.002). </span></span></span></p>

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<p style="text-align:justify"><span style="font-size:12pt"><span style="font-family:Aptos,sans-serif"><span style="font-family:&quot;Times New Roman&quot;,serif">During a median follow-up of 1.9 [0.9&ndash;3.2] years, 109 patients (39.2%) met the primary endpoint, including 82 deaths. Kaplan&ndash;Meier analysis demonstrated an improved event-free survival favouring tafamidis <span style="color:black">(HR 0.17; 95% CI 0.11&ndash;0.25; p&lt;0.001) (</span><strong><span style="color:#0070c0">central figure</span></strong><span style="color:black">). I</span>n the multivariate model adjusted for age, sex, NYHA, left ventricular ejection fraction, NT-proBNP and creatinine, tafamidis remained an independent predictor of reduced risk for the primary endpoint (HR 0.28; 95% CI 0.18-0.43; p&lt;0.001), driven by a reduction in death and cardiovascular hospitalization. These findings were consistent while assessing the cohort by NAC subgroups.</span></span></span></p>

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<p style="text-align:justify"><span style="font-size:12pt"><span style="font-family:Aptos,sans-serif"><strong><span style="font-family:&quot;Times New Roman&quot;,serif">Conclusion:</span></strong></span></span></p>

<p style="text-align:justify"><span style="font-size:12pt"><span style="font-family:Aptos,sans-serif"><span style="font-family:&quot;Times New Roman&quot;,serif">In this real-world cohort of patients with symptomatic HF due to ATTR-CM, tafamidis therapy was independently associated with a reduction in cardiovascular hospitalization or all-cause mortality. The magnitude of the benefit was comparable to that observed in the ATTR-ACT, supporting the effectiveness of applying trial-derived eligibility criteria to guide appropriate patient selection in real-world clinical practice.</span></span></span></p>