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The study aimed to demonstrate the effects of the RNA interference therapeutic, patisiran, on transthyretin-mediated (ATTR) amyloidosis. ATTR amyloidosis is caused by the buildup of transthyretin protein fibrils in the heart and other organs, leading to reduced stroke volume and decreased isovolumetric pressure-volume area over time. The study used transthoracic echocardiography and non-invasive pressure-volume techniques to assess the effects of patisiran on left ventricular (LV) stroke volume (SV) and LV mechanics. The results showed that patisiran might delay the progression of LV chamber dysfunction starting at 9 months of therapy and that decline in LV SV was driven by diminished LV capacitance in the placebo group relative to the patisiran group. The study highlights the mechanisms by which transthyretin-reducing therapies can modulate the progression of cardiac disease.
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