Am J Health Syst Pharm. 2026 Apr 7:zxag106. doi: 10.1093/ajhp/zxag106. Online ahead of print.
ABSTRACT
PURPOSE: The TAFA-CRUZ study evaluated the real-world effectiveness and safety of tafamidis 61 mg in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) and heart failure. The primary objective was to evaluate all-cause mortality, with key secondary outcomes including 30-month survival, cardiovascular-related deaths, and safety.
METHODS: This prospective, single-center observational study included 238 patients with ATTR-CM followed between 2019 and 2024 in a structured amyloidosis program. Of these patients, 140 were initiated on tafamidis 61 mg in additional to receiving supportive care guided by the CHAD-STOP strategy, while 98 received only supportive care. Baseline demographics, outcomes, and subgroup analyses, including National Amyloidosis Centre (NAC) stages, were assessed.
RESULTS: The cohort was predominantly elderly (mean age, 81.8 years) and male (80.7%). Patients treated with tafamidis were, on average, younger and less symptomatic at baseline than those in the comparator group. After a median follow-up of 21 months, treatment with tafamidis, as compared to supportive care alone, was associated with lower all-cause mortality (16.4% vs 54.1%) and cardiovascular mortality (13.6% vs 39.8%). Thirty-month survival was significantly higher in the tafamidis group (92.1% vs 51.0%; hazard ratio [HR], 0.17 [95% CI, 0.10-0.32]; P < 0.001). Survival benefits were consistent across all NAC stages, with the greatest relative effect observed in NAC stage 3. Tafamidis was well tolerated, with adverse events mostly limited to mild transient gastrointestinal symptoms and rare treatment discontinuations.
CONCLUSION: In this real-world cohort, treatment with tafamidis 61 mg was effective and safe, being associated with significantly reduced all-cause and cardiovascular mortality across all disease stages. These findings extend clinical trial evidence into routine practice and highlight the value of specialized amyloidosis programs in optimizing outcomes for patients with ATTR-CM.
PMID:41951366 | DOI:10.1093/ajhp/zxag106