Abstract
We performed an observational study on health-related quality of life (HRQoL) in patients with AL amyloidosis diagnosed between 2012 and 2017 at our institution. A total of 81 patients were included, with a median age of 64 years. The mean FACT-G (Functional Assessment of Cancer Therapy-General) total score at baseline (≤2 months from diagnosis) was 74 (±15), compared to a normative score of 80 (±18) in the general U.S. population. Significant HRQoL deficit was noted only in the functional well-being (FWB) domain of FACT-G. Using PROMIS-GH (Patient-Reported Outcomes Measurement Information System-Global Health) at baseline (n = 18), a greater deficit was noted in the global physical health (GPH) compared to global mental health (GMH) domain. FACT-FWB and PROMIS-GPH domain scores were able to significantly discriminate between revised Mayo stages. Development and validation of an amyloid-specific PRO instrument incorporating specific domains of interest is urgently needed to pursue patient-centered drug development.
Disclosure statement
No potential conflict of interest was reported by the author(s).
Author contributions
R.C. and N.S.M. designed the study, analyzed the data, wrote the first draft and approved the final version of the manuscript. L.R. performed the statistical analysis and revised the manuscript critically. J.T. maintained the patient database with clinical and demographic information. C.J.S, B.M.F. and J.V. performed patient management, revised the manuscript critically and approved the final draft of the manuscript.