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Purpose of Review <p>This review examines the recent advances in transthyretin amyloid cardiomyopathy (ATTR-CM), focusing on evolving diagnostic strategies and emerging therapeutic interventions. We sought to analyze the current state of diagnostic modalities, evaluate newly approved disease-modifying therapies, and discuss the impact of these developments on patient outcomes and clinical practice patterns.</p> Recent Findings <p>The incidence of ATTR-CM has increased dramatically due to improved recognition particularly among women and non-invasive diagnostics with bone scintigraphy utilization. Diagnostic advances include refined clinical scoring systems such as ATTR-CM and T-AMYLO as well as more widespread use of MRI. The therapeutic landscape has expanded significantly with FDA approval of three disease-modifying agents: tafamidis (TTR stabilizer), acoramidis (TTR stabilizer achieving &gt; 90% stabilization), and vutrisiran (siRNA reducing TTR by ~ 80%). SGLT inhibitors show promising results, and emerging therapies include antibody-mediated fibril removal agents and gene editing with CRISPR-Cas9.</p> Summary <p>ATTR-CM has evolved from an underdiagnosed condition to a treatable cardiomyopathy with multiple therapeutic options targeting different pathophysiological mechanisms. Early diagnosis and treatment initiation are crucial for optimal outcomes, with disease-modifying therapies showing clear mortality and hospitalization benefits. Future research should focus on comparative effectiveness studies between available therapies, optimal treatment sequencing, and addressing healthcare accessibility challenges given the high cost of current treatments.</p>

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Transthyretin Amyloid Cardiomyopathy: Diagnosis and Management

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2025年8月7日

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  • Anja Selland,
  • Jalal Agakishi,
  • Adam Devine,
  • Valmiki R Maharaj

摘要

Purpose of Review

This review examines the recent advances in transthyretin amyloid cardiomyopathy (ATTR-CM), focusing on evolving diagnostic strategies and emerging therapeutic interventions. We sought to analyze the current state of diagnostic modalities, evaluate newly approved disease-modifying therapies, and discuss the impact of these developments on patient outcomes and clinical practice patterns.

Recent Findings

The incidence of ATTR-CM has increased dramatically due to improved recognition particularly among women and non-invasive diagnostics with bone scintigraphy utilization. Diagnostic advances include refined clinical scoring systems such as ATTR-CM and T-AMYLO as well as more widespread use of MRI. The therapeutic landscape has expanded significantly with FDA approval of three disease-modifying agents: tafamidis (TTR stabilizer), acoramidis (TTR stabilizer achieving > 90% stabilization), and vutrisiran (siRNA reducing TTR by ~ 80%). SGLT inhibitors show promising results, and emerging therapies include antibody-mediated fibril removal agents and gene editing with CRISPR-Cas9.

Summary

ATTR-CM has evolved from an underdiagnosed condition to a treatable cardiomyopathy with multiple therapeutic options targeting different pathophysiological mechanisms. Early diagnosis and treatment initiation are crucial for optimal outcomes, with disease-modifying therapies showing clear mortality and hospitalization benefits. Future research should focus on comparative effectiveness studies between available therapies, optimal treatment sequencing, and addressing healthcare accessibility challenges given the high cost of current treatments.