SPC365

Clinical Profile, Treatment Selection, and Early Outcomes with TTR stabilizers to treat patients with Transthyretin Cardiac Amyloidosis: A Real-World Study

Session: Sessão de Posters 12 - Estratégias terapêuticas e desfechos em vida real na ATTR-CM
Speaker: Mariana Rocha Morato Silveira Ramos

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: Mariana Silveira Ramos; Rita Carvalho; Sérgio Maltês; Rui Gomes; Tânia Laranjeira; Gonçalo Cunha; Miguel Mendes; Carlos Aguiar; Bruno M. Rocha

<p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong><span style="font-size:12.0pt">Background:</span></strong><span style="font-size:12.0pt"><span style="font-family:&quot;Times New Roman&quot;,serif"> Transthyretin cardiac amyloidosis (ATTR-CM) is a progressive infiltrative cardiomyopathy treatable with transthyretin stabilizers (S-TTR): 1) tafamidis 61mg id, which is more broadly available and has long-term real-world data; and 2) acoramidis 712mg bid, restricted to an Early Access Program (EAP) in Portugal, with scarcer real-world evidence.</span></span></span></span></p>

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<p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong><span style="font-size:12.0pt">Objective: </span></strong><span style="font-size:12.0pt"><span style="font-family:&quot;Times New Roman&quot;,serif">To characterize the real-world local experience with S-TTR to treat ATTR-CM, comparing baseline characteristics, treatment selection, safety, and clinical outcomes.</span></span></span></span></p>

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<p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong><span style="font-size:12.0pt">Methods: </span></strong><span style="font-size:12.0pt"><span style="font-family:&quot;Times New Roman&quot;,serif">We conducted a retrospective, single-center cohort study of consecutive patients with ATTR-CM diagnosed according to the recommended algorithm. Treatment allocation reflected local protocols and EAP criteria. The primary endpoint was a composite of all-cause mortality or first cardiovascular hospitalization. </span></span></span></span></p>

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<p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong><span style="font-size:12.0pt">Results: </span></strong><span style="font-size:12.0pt"><span style="font-family:&quot;Times New Roman&quot;,serif">A total of 180 patients received S-TTR: 170 were treated with tafamidis and 10 with acoramidis. The acoramidis group was slightly younger (80 [77-90] vs. 83 [79-83] years) and included more women (30% vs. 17%), with more pronounced renal dysfunction [creatinine clearance: 28 [25&ndash;33] vs. 44 [34&ndash;60] mL/min; <em>p</em>=0.001] and higher NT-proBNP levels (3954 [1372&ndash;13200] vs. 2197 [1323&ndash;4005] pg/mL; <em>p</em>=0.340). Echocardiographic parameters were similar between groups at baseline: interventricular septal thickness (17 [15&ndash;19] vs. 17 [14&ndash;21] mm), left ventricular ejection fraction (54% [43&ndash;58] vs. 53% [48&ndash;60]), tricuspid annular plane systolic excursion (17 [13&ndash;19] vs. 17 [13&ndash;20] mm) and Perugini grading (grade 3: 63% vs. 40%; grade 2: 35% vs. 40%). </span></span></span></span></p>

<p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><span style="font-size:12.0pt"><span style="font-family:&quot;Times New Roman&quot;,serif">Acoramidis was prescribed according to the EAP: 3 patients switched from tafamidis (1 due to worsening heart failure; 2 due to progressive renal dysfunction) and 7 initiated acoramidis upfront due to chronic kidney disease (glomerular filtration &lt;25 mL/min). Patients received tafamidis and acoramidis for a median of 20 (10-35) vs. 3 (0-4) months (p&lt;0.001). During follow-up, 45 patients (25%) met an event of the primary endpoint: 33 died and 25 had a first cardiovascular hospitalization (<strong><span style="color:#0070c0">central figure</span></strong>). Thirty-month survival estimated by Kaplan&ndash;Meier for the overall cohort (n=180) was 81.7% (95% CI 75.8&ndash;87.6). The acoramidis cohort had a 100% event-free survival, with no new reported adverse events, although interpretation is limited by the short follow-up.</span></span></span></span></p>

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<p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong><span style="font-size:12.0pt">Conclusion: </span></strong><span style="font-size:12.0pt">In this real-world cohort of patients with ATTR-CM treated with S-TTR, those on acoramidis had<span style="font-family:&quot;Times New Roman&quot;,serif"> similar features compared to those on tafamidis, with the seldom exception of more pronounced renal impairment and higher NT-proBNP levels. Patients on acoramidis remained free of cardiovascular hospitalizations and mortality during follow-up. These early real-world findings support a favorable safety profile of acoramidis in EAP-selected patients unsuitable for or with disease progression while on tafamidis.</span></span></span></span></p>