Expert consensus on the monitoring of transthyretin amyloid cardiomyopathy

Abstract

Transthyretin amyloid cardiomyopathy (ATTR‐CM) is a life‐threatening condition with a heterogeneous clinical presentation. The recent availability of treatment for ATTR‐CM has stimulated increased awareness of the disease and patient identification. Stratification of patients with ATTR‐CM is critical for optimal management and treatment; however, monitoring disease progression is challenging and currently lacks best‐practice guidance. In this report, experts with experience in treating amyloidosis and ATTR‐CM developed consensus recommendations for monitoring the course of patients with ATTR‐CM and proposed meaningful thresholds and frequency for specific parameters. A set of 11 measurable features across three separate domains were evaluated: (i) clinical and functional endpoints, (ii) biomarkers and laboratory markers, and (iii) imaging and electrocardiographic parameters. Experts recommended that one marker from each of the three domains provides the minimum requirements for assessing disease progression. Assessment of cardiac disease status should be part of a multiparametric evaluation in which progression, stability or improvement of other involved systems in transthyretin amyloidosis should also be considered. Additional data from placebo arms of clinical trials and future studies assessing ATTR‐CM will help to elucidate, refine and define these and other measurements.

Baseline Characteristics and Secondary Medication Adherence Patterns Among Patients Receiving Tafamidis Prescriptions: A Retrospective Analysis Using a National Specialty Pharmacy Dispensing Database

“…An increased amount of screening and awareness of ATTR-CM is needed to provide a complete and accurate perspective of the population with ATTR-CM diagnoses. 1 , 3 Until this occurs, tafamidis as a treatment of ATTR-CM may be underutilized, resulting in patient selection biases, and thus may affect available real-world data.…”

Targeting Amyloid Fibrils by Passive Immunotherapy in Systemic Amyloidosis

“…Assessment of cardiac disease status should be part of a multiparametric evaluation. A consensus panel provided criteria of disease progression during therapy belonging to three categories [ 25 ]: (1) clinical and functional endpoints (any hospitalization for heart failure; any increase in New York Heart Association [NYHA] class; 10% decline in EQ-5D score; 40 m decrease in 6MWT); (2) biomarkers and laboratory markers (30% increase in NT-proBNP with 300 ng/L cut-off; 30% increase in high-sensitivity troponin; and (3) imaging and electrocardiographic parameters (left ventricle ejection fraction decrease > 5%; > 5 mL decrease in SV; ≥ 2 mm increase in left ventricle wall thickness; new-onset atrial fibrillation or atrioventricular block or bundle branch block; stepwise increase in diastolic dysfunction grade). The authors suggested using one marker from each category to assess response to therapy.…”

Heart transplantation in cardiac amyloidosis

“…Conversely, a partial response consistent in a reduction of 50% or more in concentrations of aberrant free light chains is not satisfactory [31] . It is known that renal dysfunction and gastrointestinal amyloid involvement may be associated with a worse prognosis and a higher rate of complications after ASCT [32] . For these reasons, it is mandatory to deeply evaluate all candidates for any gastrointestinal, hepatic, or renal involvement, including severe neuropathy or coagulopathy.…”