Patient Related Outcome Measures (Jul 2023)

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

  • O'Connor M,
  • Hsu K,
  • Broderick L,
  • McCausland KL,
  • LaGasse K,
  • Rebello S,
  • Carty M,
  • Lousada I

Journal volume & issue
Vol. Volume 14
pp. 213 – 222

Abstract

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Meaghan O’Connor,1 Kristen Hsu,2 Lynne Broderick,1 Kristen L McCausland,1 Kaitlin LaGasse,1 Sabrina Rebello,2 Michelle Carty,1 Isabelle Lousada2 1QualityMetric Incorporated LLC, Johnston, RI, USA; 2Amyloidosis Research Consortium, Newton, MA, USACorrespondence: Kristen Hsu, Amyloidosis Research Consortium, 320 Nevada Street, Suite 210, Newton, MA, 02460, USA, Tel +1-617-467-5170, Email [email protected]: 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.Methods: 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.Results: 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.Conclusion: 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.Keywords: content validity, cardiomyopathy, polyneuropathy, qualitative, clinical outcome assessment

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