Association Between Hematologic Response and Change in Health-Related Quality of Life Among Patients with Light-Chain (AL) Amyloidosis

Abstract

Purpose

The purpose of this secondary data analysis was to examine the association between hematologic response and health-related quality of life (HRQoL) among patients with light-chain (AL) amyloidosis.

Patients and Methods

Data for this secondary analysis were collected through a non-interventional, longitudinal, online self-report survey of patients with AL amyloidosis. Patients completed an initial online survey, with follow-up surveys administered 1, 6, 12, 18, and 24 months after completion of the initial survey. The online survey included an assessment of patients’ most recent self-reported hematologic response status. Eight domains and 2 summary components of HRQoL were evaluated with the SF-36v2^® Health Survey. A series of logistic regression models were used to examine the association between self-reported hematologic response at 24 months (dichotomized as new or maintained complete hematologic response; less than a complete response) and change in HRQoL from baseline to 24 months (dichotomized as meaningful worsening; improvement or preservation).

Results

For all measured domains of HRQoL except physical functioning, there was no statistically significant relationship between meaningful worsening in HRQoL and hematologic response status at 24 months. Patients without a complete hematologic response had an odds of experiencing meaningful worsening of HRQoL that was similar to that of patients with a complete hematologic response.

Conclusion

Among patients with AL amyloidosis, change in HRQoL was generally not associated with hematologic response. Achieving a complete hematologic response does not necessarily mean that a patient will experience increased or stable HRQoL. When defining treatment success, it is important to recognize that clinical markers such as hematologic response may not fully encapsulate the patient experience.

Keywords:

• patient-reported outcomes
• SF-36v2
• treatment response
• primary amyloidosis

Abbreviations

AL, amyloidosis, light-chain amyloidosis; CI, confidence interval; HRQoL, health-related quality of life; SF-36v2, SF-36v2^® Health Survey.

Ethics Approval and Informed Consent

All procedures 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 forms, protocols, surveys, and interview guide were approved by the New England Independent Review Board (# 15-355). All participants provided consent.

Acknowledgments

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

Disclosure

AC is an employee of and TPQ is a former employee of Prothena Bioscience Inc and both are stockholders of Prothena Corporation plc. AAR, KLM, and MKC are employees of QualityMetric Incorporated, LLC, and received funding from Prothena to conduct this research. QualityMetric owns the copyright to and receives licensing fees for the use of the SF-36v2^® Health Survey. The authors report no other conflicts of interest in this work.