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Background <p>Transthyretin (TTR) Cardiomyopathy (ATTR-CM) is characterized by the deposition of misfolded TTR monomers in the heart, leading to progressive heart failure. TTR-specific therapies offer a pharmacological approach to slow disease progression. However, there remains limited data on the efficacy, comparative effectiveness, and safety of these therapies. Therefore, we aim to perform a systematic review and meta-analysis of randomized controlled trials (RCTs) comparing TTR-specific therapies with placebo in patients with ATTR-CM.</p> Methods <p>We searched through Pubmed, Cochrane, and Embase databases. Our primary outcome was: (1) All Cause Mortality. We also performed a subgroup analysis comparing TTR stabilizers versus TTR knock-down therapies (RNA inhibitors and antisense oligonucleotides).</p> Results <p>Nine RCTs were included, involving 2,713 patients, of whom 1,160 (59.34%) were assigned to the TTR-specific therapies group. In the pooled analysis, TTR-specific therapies were associated with a significant reduction in all-cause mortality (RR 0.70; 95% CI 0.60, 0.83; <i>p</i> &lt; 0.01; I² = 0%), with both TTR stabilizers and knock-down therapies showing equally effective reductions (<i>p</i> = 0.97). Additionally, TTR-specific therapies improved LV longitudinal strain (SMD − 0.22; 95% CI -0.34, -0.10; <i>p</i> &lt; 0.01; I² = 17%) and reduced LV mass (SMD − 9.11&#xa0;g; 95% CI -16.4&#xa0;g, -1.82&#xa0;g; <i>p</i> = 0.01; I² = 0%).</p> Conclusion <p>This meta-analysis highlights the potential of TTR-targeting therapies as an effective option for managing ATTR-CM, with significant improvements in survival. No efficacy differences were found between TTR stabilizers and knock-down therapies.</p>

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The efficacy and safety of specific therapies for cardiac Transthyretin-mediated amyloidosis: a systematic review and meta-analysis of randomized trials

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年4月18日

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  • Alonzo Armani Prata,
  • Eric Shih Katsuyama,
  • Pedro Gabriel Scardini,
  • Ana Carolina Covre,
  • Wilson Falco Neto,
  • Julia Marques Fernandes,
  • Gabriel Scarpioni Barbosa,
  • Chris Fukunaga,
  • Rafael Petri Pinheiro,
  • Vanio L. J. Antunes,
  • Luciana Gioli-Pereira,
  • Fabio Fernandes

摘要

Background

Transthyretin (TTR) Cardiomyopathy (ATTR-CM) is characterized by the deposition of misfolded TTR monomers in the heart, leading to progressive heart failure. TTR-specific therapies offer a pharmacological approach to slow disease progression. However, there remains limited data on the efficacy, comparative effectiveness, and safety of these therapies. Therefore, we aim to perform a systematic review and meta-analysis of randomized controlled trials (RCTs) comparing TTR-specific therapies with placebo in patients with ATTR-CM.

Methods

We searched through Pubmed, Cochrane, and Embase databases. Our primary outcome was: (1) All Cause Mortality. We also performed a subgroup analysis comparing TTR stabilizers versus TTR knock-down therapies (RNA inhibitors and antisense oligonucleotides).

Results

Nine RCTs were included, involving 2,713 patients, of whom 1,160 (59.34%) were assigned to the TTR-specific therapies group. In the pooled analysis, TTR-specific therapies were associated with a significant reduction in all-cause mortality (RR 0.70; 95% CI 0.60, 0.83; p < 0.01; I² = 0%), with both TTR stabilizers and knock-down therapies showing equally effective reductions (p = 0.97). Additionally, TTR-specific therapies improved LV longitudinal strain (SMD − 0.22; 95% CI -0.34, -0.10; p < 0.01; I² = 17%) and reduced LV mass (SMD − 9.11 g; 95% CI -16.4 g, -1.82 g; p = 0.01; I² = 0%).

Conclusion

This meta-analysis highlights the potential of TTR-targeting therapies as an effective option for managing ATTR-CM, with significant improvements in survival. No efficacy differences were found between TTR stabilizers and knock-down therapies.