Key result
Real-world tafamidis is linked to ~6% 24-month mortality in ATTR-CM, outperforming pivotal trial rates.
Why the study?
Pivotal clinical trials demonstrated the efficacy of disease-modifying therapies in ATTR-CM, motivating a comparison of clinical characteristics and cardiovascular outcomes between a real-world Portuguese cohort treated with tafamidis and published pivotal trial cohorts.
How do the clinical characteristics and 24-month all-cause mortality of real-world Portuguese patients with ATTR-CM treated with tafamidis 61 mg once daily compare with those reported in pivotal clinical trials?
Cohort (n=89)
No
How do the clinical characteristics and 24-month all-cause mortality of real-world Portuguese patients with ATTR-CM treated with tafamidis 61 mg once daily compare with those reported in pivotal clinical trials?
Real-world Portuguese patients with ATTR-CM treated with tafamidis 61 mg had better baseline functional status and lower 24-month mortality (5.6%) than populations in pivotal clinical trials, supporting the real-world effectiveness of tafamidis.
Answers are grounded in this paper and cite their sources.
Lower mortality in this real-world ATTR-CM cohort may reflect earlier-stage disease; leaves open generalizability of trial benefits.
INTRODUCTION AND OBJECTIVES: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive and life-threatening disease that leads to heart failure and increased mortality. Pivotal clinical trials - ATTR-ACT, ATTRibute-CM and HELIOS-B - have provided robust evidence supporting the efficacy of TTR stabilizers (Tafamidis and Acoramidis) and the TTR-synthesis inhibitor Vutrisiran in reducing mortality and cardiovascular events. This study aims to compare the clinical characteristics and cardiovascular outcomes of a real-world Portuguese cohort of patients with ATTR-CM treated with tafamidis to those reported in pivotal clinical trials. METHODS: Single-center, observational study including consecutive patients with ATTR-CM treated with tafamidis 61 mg once a day. Clinical, laboratory, and echocardiographic data were collected and compared with published results from the ATTR-ACT, ATTRibute-CM, and HELIOS-B trials. The primary endpoint was all-cause mortality at 24 months, assessed by Kaplan-Meier survival analysis. Secondary outcomes included cardiovascular-related hospitalizations and quality of life, assessed by KCCQ-OS during follow-up, both of which were compared across studies. RESULTS: A total of 89 patients were included (76 males; 39 with hereditary ATTR-CM and 50 with wild-type ATTR-CM). The Portuguese real-world cohort was older than the ATTR-ACT and HELIOS-B study populations (mean age 78.6 vs. 74.5 vs. 77.0 years, respectively; p<0.001) and exhibited better functional capacity as assessed by New York Heart Association class compared to ATTR-ACT, ATTRibute-CM and HELIOS-B (New York Heart Association (NYHA) I: 30 vs. 9 vs. 12 vs. 15%, respectively; p<0.001). Echocardiography showed higher LVEF (55 vs. 48%, p<0.001) and better GLS (-15 vs -9%, p<0.001) compared with ATTR-ACT. NT-proBNP levels were comparable across cohorts (2639 vs. 2996 vs. 2946 vs. 2021 pg/mL, respectively; p>0.05). Hereditary ATTR was significantly more prevalent in the Portuguese cohort (44%) compared to ATTR-ACT (24%), ATTRibute-CM (10%), and HELIOS-B (11%; p<0.001), reflecting the endemic p.Val50Met mutation in Portugal. Over a follow-up of 27.4 ± 19.3 months, seven patients (7.9%) experienced heart failure hospitalizations and five patients (5.4%) died, including three cardiovascular deaths, corresponding to an annualized cardiovascular-related hospitalization rate of 0.034 events per patient-year. The 24-month all-cause mortality of 5.6% was considerably lower than reported in ATTR-ACT (24.2%), ATTRibute-CM (15.9%), and HELIOS-B (11.4%). However, KCCQ-OS scores were lower than in ATTRibute-CM and HELIOS-B (both p<0.001), likely reflecting comorbidities, and neurological involvement in hereditary cases. CONCLUSIONS: Patients receiving tafamidis 61 mg for ATTR-CM in this real-world cohort were older, had a higher prevalence of hereditary ATTR, and demonstrated better baseline functional capacity compared to those enrolled in pivotal clinical trials. These observational data provide valuable real-world evidence complementing clinical trials, supporting the use, and safety of tafamidis in this Portuguese cohort.
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Gregório et al. (2026) conducted a cohort in Transthyretin amyloid cardiomyopathy (ATTR-CM) (n=89). Tafamidis vs. Published cohorts from pivotal clinical trials (ATTR-ACT, ATTRibute-CM, and HELIOS-B) was evaluated on All-cause mortality at 24 months. Tafamidis once daily in a real-world ATTR-CM cohort yielded a 24-month all-cause mortality of 5.6%, compared with 24.2% in ATTR-ACT, 15.9% in ATTRibute-CM, and 11.4% in HELIOS-B.
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