Research Article: The Transthyretin Amyloidosis – Quality of Life (ATTR-QOL) Questionnaire: Development of a Conceptual Model and Disease-Specific Patient-Reported Outcome Measure
Introduction:
Transthyretin amyloidosis (ATTR) is a rare, debilitating disease caused by deposits of misfolded transthyretin proteins in organ systems. There are two types of ATTR: hereditary and wild-type. Hereditary ATTR (hATTR or ATTRv) includes more than genotypes and can be further subtyped into cardiomyopathy (hATTR-CM) and polyneuropathy (hATTR-PN) based on the primary organ systems affected. The most commonly observed genotype worldwide, VM, is primarily associated with symptoms of polyneuropathy and was first described…
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