ArticleCirculation reports2026
Preliminary Real-World Evidence of Clinical and Echocardiographic Changes After Vutrisiran Initiation in Patients With Variant Transthyretin Amyloid Cardiomyopathy in Non-Endemic Regions: A Single-Center Observational Study.
Article in Circulation reports, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background: Real-world data describing the clinical course after vutrisiran initiation in patients with variant transthyretin amyloid cardiomyopathy (ATTRv-CM) in non-endemic regions remain limited, particularly in Asian cohorts and in patients transitioning from patisiran. We evaluated preliminary real-world changes after vutrisiran initiation in patients with ATTRv-CM. Methods and Results: This single-center retrospective observational study included 18 patients with ATTRv-CM from the Oita-ATTR-CM cohort. Ten patients who initiated small interfering RNA therapy with vutrisiran were classified as the vutrisiran-naive group, and 8 patients who switched from patisiran to vutrisiran were classified as the vutrisiran-switch group. Changes in transthyretin (TTR), N-terminal pro B-type natriuretic peptide (NT-proBNP), troponin T, and echocardiographic parameters from baseline to 1 year were descriptively evaluated. Vutrisiran markedly reduced TTR in both groups. NT-proBNP significantly decreased in both groups, and troponin T decreased only in the vutrisiran-naive group. E/e' significantly decreased in both groups, whereas left ventricular ejection fraction, interventricular septal thickness, and left ventricular mass remained largely unchanged. During follow up, 2 patients were hospitalized for heart failure and 1 died of a non-cardiovascular cause; all events occurred in the vutrisiran-switch group. Conclusions: In this preliminary single-center real-world cohort, vutrisiran use was feasible and was accompanied by sustained TTR suppression and descriptive decreases in NT-proBNP and E/e' over 1 year. These findings should be interpreted as exploratory real-world observations rather than evidence of definitive efficacy.
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