ArticleNature medicine2026
Eplontersen with and without background transthyretin stabilizers in transthyretin amyloid cardiomyopathy: secondary analysis of a phase 3, randomized controlled trial.
Article in Nature medicine, 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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Authors and funding
24 authors.
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Abstract
Transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM) therapies include both TTR gene silencers and TTR stabilizers. The role of TTR gene silencers added to background TTR stabilizer therapy is unknown, and the effects of silencer treatment in the absence of stabilizer use are unclear. In the CARDIO-TTRansform study, 1,432 patients (n = 135 (9.4%) women and n = 1,297 (90.6%) men) with ATTR-CM were randomized (1:1) and treated with the antisense oligonucleotide eplontersen (45 mg every 4 weeks) that targets TTR or with placebo for up to 140 weeks. At baseline, 57% of the patients were taking TTR stabilizers. In the overall study, treatment with eplontersen did not reduce the primary composite endpoint. Here, in a prespecified analysis, we show that the primary endpoint (a composite of cardiovascular mortality and recurrent cardiovascular events) was modified by baseline stabilizer use (P
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Registered trials
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