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February 23, 2026European Journal of Internal MedicineOpen Access

How to monitor disease progression in ATTR amyloid cardiomyopathy: Implications for clinical practice and trial design

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Why the study?

As disease-modifying therapies for ATTR-CM become accessible, clinical indicators of disease progression are needed to guide clinical decision-making and personalize treatment strategies.

Population

Patients with transthyretin amyloid cardiomyopathy (ATTR-CM)

Design

Review

Key result

Markers such as outpatient diuretic intensification increased mortality risk by HR 1.79, NT-proBNP progression by HR 1.81, and myocardial ECV increase by HR 2.02 at 12 months in ATTR-CM patients, supporting their use as indicators of disease progression.

Authors

APAldostefano PorcariSCSarah CuddyMMMarco Metra

Discussion

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Overview

These markers may support ATTR-CM monitoring; leaves open prospective validation before guiding practice.

Key Points

  • This research aims to identify clinical indicators for monitoring disease progression in ATTR amyloid cardiomyopathy.
  • Evaluate changes in heart failure symptoms and diuretic management.
  • Analyze the 6-minute walk test and biomarker levels such as N-terminal pro-B-type natriuretic peptide.
  • Use cardiac imaging to assess structural and functional parameters.
  • Certain clinical indicators effectively track disease evolution in ATTR-CM.
  • Integrated, multiparametric approaches improve disease assessment accuracy.
  • Novel therapies may lead to disease regression even in advanced cases.

Structured PICO

P
Population
Patients with transthyretin amyloid cardiomyopathy (ATTR-CM)

Monitoring disease progression in ATTR-CM requires a multiparametric approach incorporating clinical, biomarker, and imaging parameters to guide clinical decision-making and trial design.

Limitations

  • Review article summarizing multiple studies; no new original data generated.
  • Most progression markers derived from retrospective cohorts with heterogeneous populations.
  • Limited validation across sexes, ethnic groups, and genotypes.
  • Potential confounding by non-ATTR-CM factors and therapies affecting biomarkers.
  • Variability in measurement techniques and intervals across studies.
  • Lack of universally accepted surrogate endpoints for disease progression in ATTR-CM.
  • Advanced monitoring tools not universally available limiting generalizability.

Cite This Study

Porcari et al. (2026) conducted a review in Patients with transthyretin amyloid cardiomyopathy (ATTR-CM), including wild-type and variant forms, varying stages including early diagnosis and advanced involvement. Markers such as outpatient diuretic intensification increased mortality risk by HR 1.79, NT-proBNP progression by HR 1.81, and myocardial ECV increase by HR 2.02 at 12 months in ATTR-CM patients, supporting their use as indicators of disease progression.

synapsesocial.com/papers/699beedd1c6c6bad539802a3https://doi.org/10.1016/j.ejim.2026.106773
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