Effects of Eplontersen on Cardiac Structure and Function in Patients With Transthyretin Amyloidosis With Cardiomyopathy: Insights from the CARDIO-TTRansform Trial.

Eplontersen modestly attenuated deterioration in left and right ventricular function in CARDIO-TTRansform patients with TTR amyloidosis with cardiomyopathy, especially without baseline stabilizers.

• Why it matters: CARDIO-TTRansform showed no overall significant reduction in cardiovascular outcomes, leaving uncertainty about whether TTR silencing improves cardiac structure and function.
• What they did: In a randomized trial of 1432 patients given eplontersen 45 mg every 4 weeks or placebo, echocardiograms were assessed at baseline and weeks 61, 97, and 140, with week-140 between-group comparisons and Cox/rates models linking baseline measures to outcomes.
• The result: At week 140, eplontersen reduced mean LV wall thickness increase (LSM difference -0.24 mm) and declines in LV stroke volume (1.88 mL), myocardial contraction fraction (0.88%), and average E/e' (-0.71), with improved right ventricular outflow tract velocity-time integral (0.42) versus placebo; effects were almost exclusively in patients not receiving stabilizers, with no significant effects in those receiving stabilizers.

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