Abstract Details

Title Reliability and Validity of the Transthyretin Amyloidosis – Quality of Life (ATTR-QOL) Questionnaire Impact Scales

Topic General Neurology

Presentation(s) P9 - Poster Session 9 (11:45 AM-12:45 PM)

Poster/Presentation
Number 2-009

Objective

To evaluate the structural validity, reliability, and construct validity of the ATTR-QOL Impact scales.

Background Transthyretin (ATTR) amyloidosis is a rare disease with heterogenous clinical presentation. This heterogeneity introduces challenges in the measurement of health-related quality of life. The ATTR-QOL is a disease-specific, patient-reported outcome measure developed to capture the symptoms and impacts from patients across all types of ATTR amyloidosis (i.e., hereditary and wild-type).

Design/Methods Adult patients with symptomatic ATTR amyloidosis in the United States completed the ATTR-QOL at 2 timepoints, along with additional surveys which served as criterion measures. Factor analyses and tests of differential item functioning (DIF) were conducted to confirm a hypothesized domain structure and identify redundant items. A scoring algorithm was proposed and tested. Psychometric analyses included tests of internal consistency reliability, test-retest reliability, convergent validity, and known-groups validity.

Results The analytic sample included 233 participants. Factor analysis and tests of DIF identified redundant items and supported the formation of 4 impact domains: Daily Activities, Social/Role Functioning, Emotional Wellbeing, and Physical Functioning. Results were confirmed with a secondary dataset. The proposed scoring algorithm was refined. Twelve impact items were dropped from the ATTR-QOLv2 as a result of factor and DIF analyses. Results supported the internal consistency reliability, test-retest reliability, convergent validity, and known-groups validity of the ATTR-QOL Impact scales. Patients with worse symptom severity, cardiac functioning, or unemployment due to ATTR amyloidosis had worse ATTR-QOL Impact scale scores.

Conclusions Findings support the use of the 4 ATTR-QOL Impact scales in patients with ATTR amyloidosis. This study resulted in updates to the ATTR-QOL for item reduction and the development of a scoring algorithm. Future work will be conducted in a clinical setting to explore responsiveness and establish meaningful within-patient change thresholds.

Authors/Disclosures
Sabrina Rebello, MPH PRESENTER	Mrs. Rebello has received personal compensation for serving as an employee of Amyloidosis Research Consortrium.
Kristen L. McCausland, PhD	Dr. McCausland has received personal compensation for serving as an employee of Quality Metric, an IQVIA company.
Kristen Hsu	Mrs. Hsu has received personal compensation for serving as an employee of Amyloidosis Research Consortium.
Andrew Lovley, MA	Mr. Lovley has nothing to disclose.
Isabelle Lousada	Ms. Lousada has received personal compensation for serving as an employee of Amyloidosis Research Consortium. The institution of Ms. Lousada has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion Pharmaceuticals. The institution of Ms. Lousada has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Prothena Biotechnology. Ms. Lousada has received personal compensation in the range of $500-$4,999 for serving as a Patient Advisory Panel with PCORI.
Kaitlin LaGasse	Ms. LaGasse has nothing to disclose.
Jakob B. Bjorner, MD, PhD	Dr. Bjorner has received personal compensation for serving as an employee of IQVIA Quality Metric.
Michelle Carty, PhD	Dr. Carty has nothing to disclose.

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