Tafamidis in the Contemporary Management of Transthyretin Cardiac Amyloidosis: A Systematic Review
In brief
Tafamidis linked to lower mortality and slower decline in ATTR-CM
A review of 17 studies involving 1,890 patients found that tafamidis was associated with reduced all-cause death and preservation of cardiac function compared with untreated cohorts, while echocardiographic strain and functional measures showed slower disease progression. Biomarker changes were inconsistent and safety data were limited, highlighting the need for prospective trials with uniform outcomes.
- Journal
- Journal of clinical medicine (Q1)
- Published
- 25 July 2026
- Study design
- Systematic review / meta-analysis of RCTs
- Evidence level
- Level 1, High (CEBM 1a)
- Authors
- Ameen Nasser, Mateusz Michalczak, Alexandra Malkowski, Wiktoria Małgorzata Zgoda, Jakub Michalczak, Anna Żądło, et al.
- PMID
- 42589936
- DOI
- 10.3390/jcm15155832
Why clinicians should know about it
- Picked for Biochemistry (medical) (top studies of the week, 16 August 2026): Tafamidis review includes biomarker outcomes for transthyretin amyloidosis
Abstract
Background: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive infiltrative cardiomyopathy associated with substantial morbidity and mortality. This systematic review evaluates the effects of tafamidis on echocardiographic parameters, cardiac biomarkers, functional outcomes, mortality, and safety in patients with ATTR-CM. Methods: This systematic review was conducted in accordance with PRISMA 2020 guidelines and registered in PROSPERO (CRD420261391553). PubMed, Embase, and Web of Science were searched through 20 March 2026. Eligible studies included adults with ATTR-CM treated with tafamidis and reporting, echocardiographic, biomarker, functional, mortality, or safety outcomes. Randomized controlled trials and observational studies were included. Risk of bias was assessed using the Cochrane RoB 2 tool and Newcastle-Ottawa Scale. Due to substantial heterogeneity, a narrative synthesis was performed. Results: Seventeen studies were included with a total of 1890 patients. Tafamidis treatment was potentially associated with stabilization of global longitudinal strain, preservation of functional status, and lower all-cause mortality compared with untreated or control cohorts. Biomarker findings, including N-terminal pro-B-type natriuretic peptide (NT-proBNP) and high-sensitivity cardiac troponin T (hs-cTnT), were heterogeneous and did not demonstrate a consistent pattern of improvement. Functional outcomes, including New York Heart Association (NYHA) class, 6-minute walk distance (6MWT), National Amyloidosis Center (NAC) staging, and quality-of-life measures, suggested slower clinical deterioration among treated patients. Limited available safety data indicated that tafamidis was generally well tolerated, with no major safety concerns identified. Conclusions: Current evidence may suggest that tafamidis slows disease progression and may potentially be associated with improved survival in ATTR-CM. Further prospective studies with standardized outcome reporting are needed. Evidence was limited by heterogeneity in study design, outcome reporting, and follow-up duration, with most included studies being observational.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.