The ATTR-QOL represents an important advancement in patient-centered amyloidosis research
• A single survey
• For Wild Type and Hereditary ATTR
• For use in Clinical trials and care
ATTR Quality of Life PRO
Asking the right questions to truly understand the ATTR amyloidosis patient experience.
The Transthyretin Amyloidosis Quality of Life (ATTR-QOL) questionnaire is the first comprehensive patient reported outcome (PRO) measure designed specifically for ATTR amyloidosis patients.
To date, studies focused on ATTR have relied on a patchwork of PROs designed for other conditions, leaving gaps in concept coverage, and increasing the burden on patients, who are asked to complete multiple questionnaires. The ATTR-QOL includes concepts deemed most important to track according to both experts and patients and does so using language approved by clinicians and understood by patients.
The questionnaire was developed by using a rigorous research design with methods that incorporated the voice of the patient at every step; alignment with the International Society for Quality of Life Research (ISOQOL) minimum standards for PRO development and recommendations from a 2021 review of 7 key guidance documents (including guidance from the US Food and Drug Administration [FDA]) for PRO development conducted by the Patient-Reported Outcomes Tools: Engaging Users & Stakeholders (PROTEUS) Consortium; and the inclusion of multiple clinical and research expert perspectives.
A New Era of Research
Development & Validation
Evidence of Content Validity
Followed recommendations from regulatory bodies (FDA & EMA)
Included rigorous, iterative, qualitative research with input from:
- patients
- multidisciplinary experts/clinicians
- measurement experts
- drug developers
Scoring & Psychometric Validation
Observational study of 233 patients with ATTR amyloidosis
Data were used to:
- confirm factor structure and develop scoring algorithms
- reduce potentially redundant items
- evaluate test-restest reliability & convergent/divergent validity
Next Steps
Continue to evaluate psychometric properties using other data sources (i.e. clinical trial data)
ARC, in collaboration with QualityMetric will examine:
- measurement properties in larger samples of targeted subgroups (i.e. patients with polyneuropathy)
- relationships between clinical measures and ATTR-QOL scores
- longitudinal measurement properties such as responsiveness
