Content Validation of the ATTR Amyloidosis Patient Symptom Survey: Findings from Patient and Clinician Cognitive Debriefing Interviews

Abstract

Purpose

Amyloid transthyretin (ATTR) amyloidosis is a rare, progressive, and fatal disease. The ATTR Patient Symptom Survey (ATTR-PSS) was previously developed through literature review and concept elicitation input from clinicians and patients and revised after evaluation by a patient focus group. This study further evaluated the content validity of the ATTR-PSS through qualitative cognitive debriefing interviews with clinicians and patients.

Methods

Seven clinicians and 10 patients with ATTR amyloidosis were interviewed individually regarding their overall impressions, the clarity and appropriateness of the survey, relevance of concepts measured, and comprehensiveness and comprehensibility of items and response choice sets.

Results

Clinicians acknowledged the usefulness of the ATTR-PSS in research and clinical settings. They suggested minor modifications to the survey instructions, the addition of 3 symptoms, and the transfer of 10 conditions from the symptom list to 2 separate items. Patients found the ATTR-PSS to be easy to complete and relevant to their experiences. Their feedback resulted in modification to instruction text, edits to the description of 4 symptoms, removal of 1 symptom, and addition of 2 diagnoses.

Conclusion

The findings support the content validity of the ATTR-PSS as an appropriate measure of symptom frequency, severity, and impact in patients with wild-type and hereditary ATTR amyloidosis.

Keywords:

• ATTR amyloidosis
• patient-reported outcomes
• symptom survey
• cognitive debriefing
• qualitative
• interview

Abbreviations

ASG, Amyloidosis Support Groups; ATTR, amyloid transthyretin; ATTR-PSS, ATTR Patient Symptom Survey; CM, cardiomyopathy; hATTR, amyloidosis: hereditary ATTR; ICF, informed consent form; IRB, independent review board; Norfolk QOL-DN, Norfolk Quality of Life–Diabetic Neuropathy; PN, polyneuropathy; PRO, patient-reported outcome; TTR, transthyretin; wtATTR, amyloidosis: wild-type ATTR.

Data Sharing Statement

The data sets analysed in the current study are available from the corresponding author on reasonable request.

Ethics Approval and Informed Consent

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. The informed consent form, protocol, and survey were approved by the New England Independent Review Board (IRB# 120,190,082). All patients provided written consent.

Consent for Publication

Not applicable.

Acknowledgments

We would like to thank the Amyloidosis Support Groups for their help in patient outreach, and each of the patients and clinicians who offered their time and insight for this project.

Author Contributions

All authors made substantial contributions to conception and design, acquisition of data, or analysis and interpretation of data; took part in drafting the article or revising it critically for important intellectual content; agreed to submit to the current journal; gave final approval of the version to be published; and agreed to be accountable for all aspects of the work.

Disclosure

AAR, LEB, and MKW are employees of Optum and received funding from Prothena Biosciences Inc to conduct this research. TPQ is an employee and stockholder of Prothena Biosciences Inc. The authors report no other conflicts of interest in this work.

Additional information

Funding

This research was funded by Prothena Biosciences Inc. The study sponsor was involved in research design, data interpretation, and drafting of the article.