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Breaking Barriers in ATTR-CM: Focusing in on Emerging Therapies - Episode 16

Prognostic Indicators of Disease Progression in ATTR-CM

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Panelists discuss how monitoring clinical indicators, cardiac imaging, biomarkers, and functional assessments—such as NT-proBNP, troponin, and the 6-minute walk test—helps detect progression in transthyretin amyloid cardiomyopathy (ATTR-CM), allowing for early intervention and optimal disease management.

Summary for Physicians: Prognostic Indicators and Detection of Progression in ATTR-CM

In ATTR-CM (transthyretin amyloid cardiomyopathy), identifying and monitoring progression is crucial for optimal patient management. Several prognostic indicators and methods for detecting progression include:

  1. Clinical Indicators:
    1. Worsening heart failure symptoms: Increasing shortness of breath, fatigue, and edema suggest cardiac deterioration.
    2. Arrhythmias: New-onset atrial fibrillation, sustained ventricular arrhythmias, or worsening arrhythmias signal disease progression.
    3. Decline in functional status: A reduction in exercise tolerance or worsening ability to perform daily activities.
  2. Cardiac Imaging:
    1. Echocardiography: Progression can be indicated by worsening left ventricular ejection fraction, increased wall thickness, or diastolic dysfunction.
    2. Cardiac MRI: Increased gadolinium retention (suggesting amyloid deposition) or worsening myocardial edema may indicate progression.
    3. Bone scintigraphy: In patients with bone tracer uptake, an increase in uptake may reflect more extensive amyloid deposition.
  3. Biomarkers:
    1. Elevated NT-proBNP: An increase in NT-proBNP levels indicates worsening heart failure and may suggest progression.
    2. Elevated troponin: Continuous or rising levels of cardiac troponin reflect myocardial injury and may signal disease progression.
    3. Free light chains and TTR levels: Changes in these biomarkers may indicate disease progression or response to treatment.
  4. Functional assessments:
    1. Six-minute walk test: A decline in the distance walked may reflect worsening functional status.
    2. Quality-of-life measures: Tools assessing functional capacity and patient-reported symptoms, such as the Kansas City Cardiomyopathy Questionnaire, can provide insight into disease progression.
  5. Other factors:
    1. TTR stabilization therapies: Lack of response or disease stabilization despite treatment may signal progression.
    2. Systemic involvement: Worsening symptoms of other organ involvement (eg, neuropathy, carpal tunnel syndrome) may also be indicative of disease progression.

In summary, progression in ATTR-CM is defined by a combination of clinical, imaging, biomarker, and functional assessments. Monitoring these factors regularly helps physicians detect progression early, enabling timely interventions to manage the disease effectively.

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