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Impact of Tafamidis on Health-Related Quality of Life in Patients with Transthyretin Amyloid Cardiomyopathy: A Comparative Study Between ATTRwt and ATTRh
Session:
Sessão de Posters 12 - Estratégias terapêuticas e desfechos em vida real na ATTR-CM
Speaker:
Rodrigo Malveiro Afonso
Congress:
CPC 2026
Topic:
F. Valvular, Myocardial, Pericardial, Pulmonary, Congenital Heart Disease
Theme:
17. Myocardial Disease
Subtheme:
17.2 Myocardial Disease – Epidemiology, Prognosis, Outcome
Session Type:
Posters Eletrónicos
FP Number:
---
Authors:
Rodrigo Afonso; Marta Vilela; Daniel Inácio Cazeiro; Diogo Ferreira; João Cravo; Catarina Campos; Isabel Conceição; Fausto J. Pinto; Dulce Brito; João R. Agostinho
Abstract
<p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><span style="color:#000000"><strong>Introduction</strong></span></span></span><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><span style="color:#000000">: In the ATTR-ACT trial, tafamidis showed a positive impact on quality of life (QoL) at 30 months, as demonstrated by improvements in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) score in patients with TTR amyloid cardiomyopathy (ATTR-CM). However, its impact on patient-reported outcomes was not evaluated separately between wild type ATTR (ATTRwt) and hereditary (ATTRh).</span></span></span></p> <p><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><span style="color:#000000"><strong>Aim</strong>: To compare the impact of tafamidis 61mg on health-related QoL in patients with ATTR-CM, as evaluated by changes in the KCCQ-OS score in patients with ATTRwt and ATTRh.</span></span></span></p> <p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><span style="color:#000000"><strong>Methods</strong>: A single center observational study was conducted. Patients were categorized into two groups: ATTRwt and ATTRh. All patients completed the 23-item KCCQ-OS during the last follow-up (Fup) visit.</span></span></span></p> <p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><span style="color:#000000"><strong>Results</strong>: 37 patients with ATTRwt and 39 patients with ATTRh were included (66 males). ATTRh patients were younger (72.5±2 vs. 83.6±0.9 years; p<0.001) and developed symptoms earlier (65.8±1.9 vs. 80.6±1.1 years, p=0.003) when compared to ATTRwt. At baseline, ATTRwt patients presented with more advanced disease, showing significantly higher NT-proBNP levels (3096.1±3076.5 vs. 1694.2±3958.9 pg/mL; p=0.04), a higher proportion of patients in NYHA functional class II or III (p<0.001), increased left ventricular hypertrophy (17.4±2.8 vs. 15.1±4.1; p=0.005), and higher diuretic dose (29.2±26.5 vs. 22.6±31.6; p<0.001).</span></span></span></p> <p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><span style="color:#000000">During a follow-up of 27.4±2.1 months, peripheral polyneuropathy was the predominant symptom in ATTRh patients (77% vs. 0%), while HF predominated in ATTRwt patients (64% vs. 81%). More patients in the ATTRwt group improved to classes I or II, whereas in the ATTRh group, patients’ functional capacity worsened - Table 1. There were no significant changes in NTproBNP levels (p=0.49) or diuretic dose (p=0.25) over the Fup period between the two groups. KCCQ-OS scores were comparable between groups (ATTRh 50.9±4.5 vs. ATTRwt 55.9±4.2, p=NS), as were scores for physical limitations (ATTRh 51.8±6.7 vs. ATTRwt 44.7±5.9, p=NS), symptoms (ATTRh 60.9±5.9 vs. ATTRwt 66.1±5.6, p=NS), QoL (ATTRh 53.4±6.2 vs. ATTRwt 55.6±5.6, p=NS), and social limitations (ATTRh 39.7±5.8 vs. ATTRwt 42.4±5.6, p=NS). However, considering the younger age of ATTRh patients, the disease likely imposes a relatively greater burden across these domains. In a gender-based sub-analysis, women with ATTRh had lower KCCQ-OS scores for social domain (p=0.02), while men with ATTRwt showed better overall KCCQ scores (p=0.023), as well as higher scores in QoL (p=0.008) and social domains (p<0.001)</span></span></span></p> <p><br /> <span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><span style="color:#000000"><strong>Conclusion</strong>: Tafamidis demonstrated a comparable impact on health-related quality of life, as assessed by KCCQ-OS scores, in patients with ATTRwt and ATTRh. Despite similar overall scores, the younger age and earlier onset of symptoms in ATTRh patients suggest a relatively greater perceived burden of the disease in this subgroup.</span></span></span></p>
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