Real-world evidence shows that new targeted therapies significantly reduce mortality in patients with transthyretin amyloid cardiomyopathy (ATTR-CM), highlighting the importance of early diagnosis, according to a recently published study in ESC Heart Failure.
ATTR-CM was once perceived as a rare untreatable condition. However, it is now recognized as a significant cause of heart failure. As diagnostic awareness increases, the number of identified cases has surged. Furthermore, the emergence of targeted therapies, including TTR protein stabilizers and gene silencers, has shifted the therapeutic landscape dramatically, offering not just symptom relief but potential survival benefits.
Randomized clinical trials have previously demonstrated that therapies like tafamidis, patisiran, vutrisiran and acoramidis improve quality of life and reduce hospitalizations in patients with ATTR-CM. Despite this, a systematic evaluation integrating both clinical trials and real-world data is lacking.
In this context, the authors aimed to close that gap by conducting a meta-analysis of 10 studies involving 5,203 patients.
The authors included studies that reported on all-cause mortality and cardiovascular hospitalizations, with data pooled from interventions involving tafamidis, patisiran, vutrisiran and acoramidis. The primary outcome was all-cause mortality, and cardiovascular hospitalizations served as a secondary endpoint. Results from previous epidemiological studies on 18,238 untreated ATTR-CM patients were used as a benchmark for estimating treatment impact.
They found that targeted therapies reduced all-cause mortality by 39%. Cardiovascular hospitalizations were also significantly lower in the treated group, though this varied more across different studies.
Applying these findings to real-world mortality data, the researchers calculated that only 10 patients need to be treated to prevent one death over two years, and just five patients need to be treated to prevent one death over five years, highlighting a substantial long-term benefit.
The authors emphasized that the magnitude of benefit was consistent across different treatment types and patient subgroups, although patient-level variability may influence outcomes. Importantly, they stressed that treatment should begin as early as possible in the disease course to maximize benefit, particularly in patients with preserved functional status.
“Early diagnosis and initiation of treatment are critical to maximize the clinical benefits and prolong patient survival,” the authors concluded.
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