28Jan 2023
Effect of Patisiran on Stroke Volume in Hereditary Transthyretin‐Mediated Amyloidosis: Insights from Pressure‐Volume Analysis of the APOLLO Study

Effect of Patisiran on Stroke Volume in Hereditary Transthyretin‐Mediated Amyloidosis: Insights from Pressure‐Volume Analysis of the APOLLO Study

Transthyretin‐mediated (ATTR) amyloidosis is caused by deposition of transthyretin protein fibrils in the heart, nerves, and other organs. Patisiran, an RNA interference (RNAi) therapeutic that inhibits hepatic synthesis of transthyretin, was approved for the treatment of hereditary ATTR amyloidosis with polyneuropathy based on the phase III APOLLO study. This study used left ventricular (LV) stroke volume (SV) to quantify LV function overtime and non‐invasive pressure‐volume techniques to delineate the effects of patisiran on LV mechanics in the prespecified cardiac subpopulation of the APOLLO study. From the study results, it was concluded that Patisiran might delay the progression of LV chamber dysfunction starting at 9 months of therapy. These data elucidate the mechanisms by which transthyretin reducing therapies modulate the progression of cardiac disease and need to be confirmed in ongoing phase III trials.

  • #cardiology

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