The Transthyretin Amyloidosis - Quality of Life (ATTR-QOL) Questionnaire: Development of a Conceptual Model and Disease-Specific Patient-Reported Outcome Measure

• Published in: Patient related outcome measures 2023-01, Vol.14, p.213-222
• Main Authors: O'Connor, Meaghan, Hsu, Kristen, Broderick, Lynne, McCausland, Kristen L, LaGasse, Kaitlin, Rebello, Sabrina, Carty, Michelle, Lousada, Isabelle
• Format: Article
• Language: English
• Subjects: Amyloidosis; Analysis; Cardiology; cardiomyopathy; clinical outcome assessment; content validity; Measurement; Medical research; Medicine, Experimental; Original Research; Patient outcomes; polyneuropathy; qualitative; Surveys
• ISSN: 1179-271X; 1179-271X
• DOI: 10.2147/PROM.S411721

Patients with transthyretin amyloidosis (ATTR) experience a wide variety of symptoms and impacts on health-related quality of life (HRQoL). However, the lack of an ATTR-specific patient-reported outcome (PRO) measure has made consistent measurement of HRQoL in ATTR challenging. This paper describes the development of a conceptual model and subsequent content for the Transthyretin Amyloidosis - Quality of Life Questionnaire (ATTR-QOL), an ATTR-specific PRO measure. This was a cross-sectional, non-interventional, US-based study. The study design included three stages: 1) a targeted literature review followed by qualitative data collection with patients and experts; 2) development of a conceptual model and PRO measure; and 3) review of the PRO measure using a modified Delphi method, translatability assessment, and interviews with patients and experts. Revisions were made to the measure after each round of review. Forty-four patients and 29 experts participated in this study. The conceptual model included two primary concepts of interest: symptoms (cardiac, neuropathic-peripheral, neuropathic-autonomic, and other) and impacts (eg, physical, role, and mental/emotional functioning). Seventy-two items were created (32 symptoms; 40 impacts) to align with the model. A recall period of one month was selected based on participant input. The ATTR-QOL was created with significant patient involvement and guidance from a multidisciplinary group of experts. The mix of patient and clinical perspectives helped to ensure a balanced representation of all relevant disease experiences and clinical specialties. With further refinement from psychometric testing, the ATTR-QOL will provide a standard, comprehensive measure for all ATTR-specific research including both clinical trials and clinical practice.