An Important Instrument for the ATTR Community
The Amyloidosis Research Consortium (ARC) spearheaded the effort to develop the ATTR-QOL in response to the needs and requests of the ATTR research community. QualityMetric provided the technical expertise to develop and evaluate the measure, collaborating with experts, clinicians, researchers, patients, patient advocates and drug developers and ensuring its alignment with current best practices in COA measure development. The resulting questionnaire provides researchers and clinicians with a standard measure for assessing the patient experience of ATTR; it can be used to inform treatment plans, better understand the natural history of this rare disease or evaluate treatment efficacy or effectiveness.
The ATTR-QOL has two primary modules that assess symptoms and impacts and a third, optional module that provides a standardized way of assessing ATTR type and 11 common comorbidities. It was developed to be used in clinical trials and research settings as well as in clinical practices and is unique in that it includes questions relevant to both primary manifestations of ATTR—polyneuropathy and cardiomyopathy—making it relevant across ATTR types (hATTR/ATTRv and ATTRwt).
Historically, research in this rare disease relied on a patchwork of existing questionnaires created for other conditions with similar symptomatologies, with some focused on the heart and others focused on neuropathies. The disease-specific ATTR-QOL eliminates the need for researchers to score and interpret multiple PRO surveys created for other conditions, reduces the likelihood of missing important symptoms and impacts and lessens the reporting burden on patients, who can complete one questionnaire instead of several.
