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Development of a conceptual model of patient-reported outcomes in light chain amyloidosis: a qualitative study

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Abstract

Background

Light chain (AL) amyloidosis is a plasma cell neoplasm associated with high early mortality and severe morbidity that can cause severe disability. We explored the impact of AL amyloidosis on symptoms and well-being from the perspectives of patients and health care providers who regularly care for AL patients. We intended to develop a conceptual understanding of patient-reported outcomes in AL amyloidosis to identify the context of use and concept of interest for a clinical outcome assessments tool in this disease.

Method

Twenty patients and ten professionals were interviewed. Patient interviews captured the spectrum of amyloidosis experience including time from diagnosis, type of organ involvement, and presence and type of treatment received. Interviews with professionals included physicians, advanced practice providers, registered nurse, and a patient advocate; these interviews covered similar topics.

Results

The impact of AL amyloidosis on patients’ life was multidimensional, with highly subjective perceptions of normality and meaning. Four major themes from patients and experts included diagnosis of AL amyloidosis, living with AL amyloidosis, symptom burden, and social roles. Barriers to patient-reported outcomes data collection in patients were additionally explored from experts. The themes provide a comprehensive understanding of the important experiences of symptom burden and its impact on daily life from AL amyloidosis patients’ and from the perspectives of professionals who care for patients with AL amyloidosis.

Conclusion

These findings further the conceptual understanding and identification of a preliminary model of concept of interest for development of a clinical outcome assessments tool for AL amyloidosis.

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Acknowledgements

The authors acknowledge Ms. Claire Piehowski’s contribution toward data management and coding assistance. Financial support for this study was provided by a K23 HL141445 Mentored Career Development Award. The funding agreement ensured the authors’ independence in designing the study, interpreting the data, writing, and publishing the report.

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Authors and Affiliations

  1. Division of Hematology/Oncology, Medical College of Wisconsin, Milwaukee, WI, 53226, USA

    Anita D’Souza, Judith Myers, Rachel Cusatis, Angela Dispenzieri, Muriel Finkel, Julie Panepinto & Kathryn E. Flynn

Authors
  1. Anita D’Souza
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  2. Judith Myers
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  3. Rachel Cusatis
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  4. Angela Dispenzieri
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  5. Muriel Finkel
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  6. Julie Panepinto
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  7. Kathryn E. Flynn
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Contributions

Conception/design: AD’S, KEF. Provision of study materials or patients: AD’S, MF. Collection and/or assembly of data: AD’S, JM, RC, KEF. Data analysis and interpretation AD’S, JM, RC, AD, MF, JP, KEF. Manuscript writing: AD’S, JM, RC, AD, MF, JP, KEF. Final approval of manuscript: AD’S, JM, RC, AD, MF, JP, KEF.

Corresponding author

Correspondence to Anita D’Souza.

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D’Souza, A., Myers, J., Cusatis, R. et al. Development of a conceptual model of patient-reported outcomes in light chain amyloidosis: a qualitative study. Qual Life Res 31, 1083–1092 (2022). https://doi.org/10.1007/s11136-021-02943-w

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  • DOI: https://doi.org/10.1007/s11136-021-02943-w

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