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Introduction <p>Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Tafamidis demonstrated efficacy in ATTR-CM clinical trials; however, real-world disease outcomes are not thoroughly characterized. We examined real-world outcomes among patients with wild-type (ATTRwt-CM) and variant (ATTRv-CM) ATTR-CM treated with tafamidis, the only approved treatment at the time of the study.</p> Methods <p>This retrospective observational study analyzed Komodo Healthcare Map® data (1/1/2016‒6/30/2024) for tafamidis-treated patients with ATTR-CM. Outcomes included all-cause hospitalization, cardiovascular-related hospitalization (CVH), heart failure (HF)-related hospitalization, outpatient worsening HF (OWHF) with oral diuretic intensification, and mortality. Subgroup analyses examined outcomes by ATTR-CM type and N-terminal pro-B-type natriuretic peptide (NT-proBNP)/B-type natriuretic peptide (BNP) baseline levels.</p> Results <p>Among 3239 tafamidis-treated patients (mean age 77.2&#xa0;years; 75.9% male; 83.0% ATTRwt-CM; 11.7% ATTRv-CM), the cumulative incidence of first all-cause hospitalization was 22% at 6&#xa0;months and 36% at 12&#xa0;months, and that of first CVH was 22% and 35%, respectively. Median time to first CVH was 699&#xa0;days. The cumulative incidence of OWHF with oral diuretic intensification was 22% at 6&#xa0;months and 33% at 12&#xa0;months. Mortality was 12.0% over the 5-year follow-up, and 6.2% at 12&#xa0;months. The cumulative incidence of the composite endpoint (CVH, OWHF, or death) was 37% within 6&#xa0;months and 53% within 12&#xa0;months. In the subgroup with NT-proBNP/BNP baseline measurements (<i>n</i> = 412), patients with high NT-proBNP (&gt; 3000&#xa0;pg/mL, or BNP &gt; 600&#xa0;pg/mL) had worse outcomes, including a higher cumulative incidence of first CVH (51% vs. 27%) and higher mortality (9.7% vs. 4.1%) at 12&#xa0;months.</p> Conclusions <p>In this large real-world cohort of tafamidis-treated patients, the cumulative incidences of hospitalization and worsening HF were substantial regardless of ATTR-CM subtype. Elevated NT-proBNP/BNP at baseline was associated with worse outcomes. These findings characterize the burden of disease outcomes in tafamidis-treated patients and underscore ongoing unmet needs in ATTR-CM management.</p>

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Real-World Outcomes Among Patients in the United States Receiving Tafamidis for Transthyretin Amyloid Cardiomyopathy

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  • 发表日期：

    2025年12月19日

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  • Daniel P. Judge,
  • Margarita Udall,
  • Andrew M. Rosen,
  • Neil Lamarre,
  • Elizabeth Nagelhout,
  • Hanh Dung Dao,
  • Mathew S. Maurer

摘要

Introduction

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Tafamidis demonstrated efficacy in ATTR-CM clinical trials; however, real-world disease outcomes are not thoroughly characterized. We examined real-world outcomes among patients with wild-type (ATTRwt-CM) and variant (ATTRv-CM) ATTR-CM treated with tafamidis, the only approved treatment at the time of the study.

Methods

This retrospective observational study analyzed Komodo Healthcare Map® data (1/1/2016‒6/30/2024) for tafamidis-treated patients with ATTR-CM. Outcomes included all-cause hospitalization, cardiovascular-related hospitalization (CVH), heart failure (HF)-related hospitalization, outpatient worsening HF (OWHF) with oral diuretic intensification, and mortality. Subgroup analyses examined outcomes by ATTR-CM type and N-terminal pro-B-type natriuretic peptide (NT-proBNP)/B-type natriuretic peptide (BNP) baseline levels.

Results

Among 3239 tafamidis-treated patients (mean age 77.2 years; 75.9% male; 83.0% ATTRwt-CM; 11.7% ATTRv-CM), the cumulative incidence of first all-cause hospitalization was 22% at 6 months and 36% at 12 months, and that of first CVH was 22% and 35%, respectively. Median time to first CVH was 699 days. The cumulative incidence of OWHF with oral diuretic intensification was 22% at 6 months and 33% at 12 months. Mortality was 12.0% over the 5-year follow-up, and 6.2% at 12 months. The cumulative incidence of the composite endpoint (CVH, OWHF, or death) was 37% within 6 months and 53% within 12 months. In the subgroup with NT-proBNP/BNP baseline measurements (n = 412), patients with high NT-proBNP (> 3000 pg/mL, or BNP > 600 pg/mL) had worse outcomes, including a higher cumulative incidence of first CVH (51% vs. 27%) and higher mortality (9.7% vs. 4.1%) at 12 months.

Conclusions

In this large real-world cohort of tafamidis-treated patients, the cumulative incidences of hospitalization and worsening HF were substantial regardless of ATTR-CM subtype. Elevated NT-proBNP/BNP at baseline was associated with worse outcomes. These findings characterize the burden of disease outcomes in tafamidis-treated patients and underscore ongoing unmet needs in ATTR-CM management.