Evidence map›Paper›PMID 40299173›Full record

ArticleJournal of patient-reported outcomes2025

Reliability and validity of the Transthyretin Amyloidosis - Quality of Life (ATTR-QOL) Questionnaire impact scales.

Andrew Lovley, Kristen Hsu, Kaitlin LaGasse, Isabelle Lousada, Kristen L McCausland, Michelle K Carty, Sabrina Rebello, Jakob B Bjorner

Abstract readValidation Study
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of patient-reported outcomes, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Andrew LovleyIQVIA Quality Metric, 225 Dyer St, 2nd Floor, Providence, RI, 02903, USA.ORCID http://orcid.org/0000-0001-9118-0673
Kristen HsuAmyloidosis Research Consortium, 320 Nevada Street, Suite 210, Newton, MA, 02460, USA. khsu@arci.org.ORCID http://orcid.org/0000-0003-2106-3652
Kaitlin LaGasseIQVIA Quality Metric, 225 Dyer St, 2nd Floor, Providence, RI, 02903, USA.ORCID http://orcid.org/0000-0001-9772-5822
Isabelle LousadaAmyloidosis Research Consortium, 320 Nevada Street, Suite 210, Newton, MA, 02460, USA.ORCID http://orcid.org/0000-0002-3506-0359
Kristen L McCauslandIQVIA Quality Metric, 225 Dyer St, 2nd Floor, Providence, RI, 02903, USA.ORCID http://orcid.org/0000-0003-4727-1046
Michelle K CartyIQVIA Quality Metric, 225 Dyer St, 2nd Floor, Providence, RI, 02903, USA.ORCID http://orcid.org/0000-0003-2450-7693
Sabrina RebelloAmyloidosis Research Consortium, 320 Nevada Street, Suite 210, Newton, MA, 02460, USA.ORCID http://orcid.org/0000-0002-0201-4030
Jakob B BjornerIQVIA Quality Metric, 225 Dyer St, 2nd Floor, Providence, RI, 02903, USA.ORCID http://orcid.org/0000-0001-7033-8224

Funding

Alnylam Pharmaceuticals Alnylam PharmaceuticalsAmyloidosis Research Consortium Amyloidosis Research ConsortiumIonis Pharmaceuticals Ionis PharmaceuticalsPfizer PfizerProthena Prothena
6 · The paper itself

Abstract

backgroundThe diversity of disease phenotypes associated with transthyretin (ATTR) amyloidosis poses challenges for measurement of health outcomes. The Transthyretin Amyloidosis - Quality of Life (ATTR-QOL) Questionnaire is a disease-specific patient-reported outcome (PRO) measure of the symptoms and impacts of ATTR amyloidosis. The objective of this study was to evaluate the structural validity, reliability, and construct validity of the ATTR-QOL Impact scales. METHODOLOGY: This was a non-interventional, online survey study of adults with self-reported diagnosis of symptomatic ATTR amyloidosis. The survey included the ATTR-QOL and additional criterion measures. A scoring algorithm was proposed and tested. Factor structure, differential item functioning, and psychometric properties were explored.

resultsThe analytic sample included 233 patients. Satisfactory fit was found for a 4-factor model of disease impacts (including Daily Activities, Physical Functioning, Social/Role Functioning, and Emotional Wellbeing) and a scoring algorithm was developed. Twelve impact items were dropped from the ATTR-QOLv2 as a result of factor and differential item functioning analyses. Each scale showed evidence of satisfactory internal consistency reliability (Cronbach's α range = 0.85-0.97) and test-retest reliability at 1 week (intraclass coefficient range = 0.84-0.97). Convergent validity was supported by correlations ≥ 0.30 between ATTR-QOL Impact scale scores and other PRO measures of related constructs. The ATTR-QOL Impact scales showed greater impact among patients with worse symptom severity, cardiac functioning, or unemployment due to ATTR amyloidosis (all p < 0.05), supporting known-groups validity.

conclusionThe ATTR-QOL is a reliable and valid measure of impacts meaningful to patients with ATTR amyloidosis. This study resulted in updates to the ATTR-QOL for item reduction and the development of a scoring algorithm. Ongoing studies are collecting data to evaluate the symptom scales of the ATTR-QOL.

Indexed as

Amyloid Neuropathies, FamilialPatient Reported Outcome MeasuresQuality of LifeActivities of Daily LivingAdultAgedAged, 80 and overFemaleHumansMaleMiddle AgedPsychometricsReproducibility of ResultsSurveys and QuestionnairesConstruct validityFactor structureHealth outcomesImpactsItem reductionPatient-reported outcome (PRO)Psychometric validationScoring developmentTransthyretin (ATTR) amyloidosis

Identifiers

PMID40299173
PMCPMC12040782

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.