Patient - Reported Burden of Hereditary Transthyretin Amyloidosis on Functioning and Well - Being

Key Information
Source
Journal of Patient-Reported Outcomes
Year
2021
summary/abstract

Hereditary transthyretin (hATTR) amyloidosis is a rare, systemic, progressive, and life-threatening disease in which transthyretin proteins misfold and aggregate as insoluble amyloid deposits, disrupting nervous, cardiac, gastrointestinal, and other organ tissues. There are limited available data about the experience of patients living with hATTR amyloidosis. This study used a qualitative, non-interventional design to explore the humanistic burden of hATTR amyloidosis from the patient's perspective. Patients were asked to describe their experiences living with the condition, including symptoms and disease-related impacts on functioning and well-being, work, and activities of daily living (ADLs). Interviews were transcribed verbatim and analyzed for key concepts using a grounded theory approach.

Abstract Source
https://jpro.springeropen.com/articles/10.1186/s41687-020-00273-y
DOI
10.1186/s41687-020-00273-y
Authors
Andrew Lovley, Kimberly Raymond, Spencer D. Guthrie, Michael Pollock, Vaishali Sanchorawala, Michelle K. White.
Organisation
QualityMetric Incorporated, LLC, USA; Aurora Bio, USA; Akcea Therapeutics, USA; Amyloidosis Center, Boston University, USA