A mini review of Patisiran's efficacy in the management of transthyretin cardiac amyloidosis

Abstract

This mini-review provides a narrative analysis of the emerging therapeutic potential of Patisiran in managing Transthyretin Cardiac Amyloidosis (ATTR-CA). ATTR-CA, characterized by the deposition of misfolded transthyretin proteins in cardiac tissue, leads to progressive heart failure, significantly impacting affected individuals' quality of life and prognosis. Current treatment options for ATTR-CA are limited, necessitating the exploration of novel interventions like Patisiran. Patisiran, an RNA interference therapeutic, targets hepatic transthyretin protein production, thereby reducing amyloid deposits. While proven effective and safe in treating ATTR amyloidosis with polyneuropathy, its impact on cardiac manifestations is less studied. Positive outcomes include improved health status, enhanced quality of life, and preservation of functional capacity. Studies demonstrate sustained benefits, highlighting the potential for long-term positive effects. Reductions in cardiac amyloid burden and regression and prevention of deterioration in various cardiac parameters suggest a potential disease-modifying effect. Despite promising results, infusion-related reactions and adverse events necessitate careful consideration of long-term tolerability. Overall, Patisiran emerges as a promising intervention, offering hope for improved patient outcomes in the complex landscape of ATTR-CA management.