Predictor of Amyloidosis Outcomes?
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A biomarker is defined by the National Institutes of Health Biomarkers Definitions Working Group as “a characteristic that is objectively measured and evaluated as an indicator of normal biological processes, pathogenic processes, or pharmacologic responses to a therapeutic intervention.”1 Several biomarkers have been studied in recent years; however, few are used in clinical practice. According to the US Preventive Services Task Force, the potential impact of a novel risk factor is based on the following: (1) its predictive ability, (2) its prevalence in the target population, (3) the number of intermediate-risk individuals who are reclassified as high risk when the risk factor is applied, and (4) the net benefit (benefits minus harms) that would accrue to these high-risk individuals if they were managed according to guidelines for high-risk patients.2
Amyloidosis involves the transformation of a precursor protein into an insoluble extracellular fibril that interferes with the function of the organs affected (Figure). The most common precursors include immunoglobulin light chains (AL amyloidosis), wild-type transthyretins (ATTRwt), and genetically mutated transthyretins (ATTRm). The field of amyloidosis continues to develop, and biomarkers have been used to estimate prognosis, guide management, and assess response to treatment. In AL amyloidosis, serum cTn (cardiac troponins) and NT-proBNP (N-terminal pro-brain natriuretic peptide) are the most studied biomarkers. In combination with other hematologic markers, they are used to classify patients into prognostic stages and to determine eligibility for stem cell transplantation, resulting in …