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A post hoc analysis of the CARDIO-TTRansform trial found that eplontersen monotherapy — without baseline TTR stabilizers — preserved health status in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) over nearly three years. Patients on eplontersen alone showed significantly less decline in quality-of-life scores compared to placebo, while those also taking TTR stabilizers saw no meaningful benefit. The findings spotlight the importance of treatment sequencing in ATTR-CM care.
A post hoc analysis of the CARDIO-TTRansform trial — the largest randomized trial in ATTR-CM to date — found that eplontersen (Wainua, Ionis/AstraZeneca) monotherapy significantly preserved health-related quality of life in patients with transthyretin amyloidosis with cardiomyopathy over 140 weeks. Presented at the Heart Failure Society of America Annual Scientific Meeting, the analysis showed that patients on eplontersen alone experienced meaningfully less decline in Kansas City Cardiomyopathy Questionnaire (KCCQ) scores compared to placebo — spanning symptoms, physical function, and quality of life.
However, patients who were also on baseline TTR stabilizers saw no significant benefit from adding eplontersen, suggesting that the combination may blunt the drug's effectiveness. This nuance is clinically important, as a large proportion of ATTR-CM patients are already on stabilizers like tafamidis.
Key Takeaways:
Why it matters: While eplontersen didn't reduce CV death or hospitalizations in the primary trial, these findings suggest it may meaningfully help patients feel and function better — particularly those not already on a TTR stabilizer, making treatment sequencing a key consideration for clinicians.
