The burden of amyloid light chain amyloidosis on health-related quality of life

Abstract

BackgroundLight chain (AL) amyloidosis is a rare disease characterized by misfolded amyloid protein deposits in tissues and vital organs, and little is known about the burden of AL amyloidosis on health-related quality of life. This study aimed to quantify the burden of AL amyloidosis in terms of health-related quality of life in a diverse, community-based sample of AL amyloidosis patients.ResultsThe SF-36v2® Health Survey (SF-36v2), a widely used generic measure of health-related quality of life (using physical and mental summary scales and subscales assessing eight aspects of functioning and well-being), was administered as an online survey of AL amyloidosis patients with AL amyloidosis (ClinicalTrials.gov, NCT02574676; n = 341). Compared with adjusted general population sample norms, health-related quality of life of AL amyloidosis patients was significantly worse across all SF-36v2 scales and summary measures based on analysis of variance (p < 0.05 for all). The largest decrement in AL amyloidosis patients was related to General Health (Δ = 9.7; p < 0.001). With the exception of Bodily Pain and Mental Health, differences were also clinically meaningful based on established clinically minimal important differences. The burden of AL amyloidosis overall and in key subgroups tended to be greater on physical health than on mental health. Stratified analyses indicated additional burden among patients with recently diagnosed disease and those with cardiac involvement than among their respective counterparts.ConclusionUnderstanding the burden of AL amyloidosis highlights the unmet need for treatment, helps physicians identify ancillary treatments and services geared towards improving patients’ functioning, well-being, and overall health-related quality of life. These findings also help to support the use of health-related quality of life end points as important outcome measures in current and future treatment studies.Trial registrationClinicalTrials.gov, NCT02574676. Registered October 5, 2015.

A longitudinal evaluation of health‐related quality of life in patients with AL amyloidosis: associations with health outcomes over time

“…The impact of AL amyloidosis and its treatments on HRQoL has not been studied extensively. Evidence from cross‐sectional studies in AL amyloidosis shows broad deficits in functioning and well‐being in both treatment‐naïve patients and in heterogeneous groups of patients with varied disease severity and treatment history (Seldin et al , ; Caccialanza et al , ; Bayliss et al , ). Only one study has reported on longitudinal assessments of HRQoL and the results indicated that greater pre‐ and post‐treatment HRQoL, as measured by the SF‐36 ® Health Survey (SF‐36), is associated with reduced risk of mortality among patients who received HDM/SCT treatments (Seldin et al , ).…”

Associations between Health-Related Quality of Life and Self-Reported Emergency Room Department Visits and Inpatient Hospitalizations: Insights from a Secondary Data Analysis of Patients with Light-Chain (AL) Amyloidosis

“…Secondary data analyses were completed using data drawn from the AL Amyloidosis Patient Health-Related Quality of Life Study [1], a longitudinal, non-interventional observational study of patients with AL amyloidosis ( n = 341) [11]. Patients were recruited for this study in the October to December of 2015 with the assistance of two patient advocacy groups (the Amyloidosis Support Groups and the Amyloidosis Foundation) using online recruitment strategies (i.e., study announcements on the patient advocacy websites and social media platforms).…”

“…While a variety of predictors of survival have been identified in patients with AL amyloidosis, including clinical assessments and biomarkers [5], less research has been conducted to explore other relevant outcomes, such as HCRU. Previous studies in other patient groups have shown a link between health-related quality of life (HRQoL) and HCRU [6–10], and certain measures of HRQoL have been shown to be sensitive to AL amyloidosis disease severity, suggesting candidates for a potential predictor of HCRU in AL amyloidosis [11].…”

Cardiac biomarkers and health‐related quality of life in patients with light chain (AL) amyloidosis

“…We conducted a cross‐sectional analysis to examine the relationship between NT‐proBNP and HRQoL in a community‐based sample of patients with AL amyloidosis (for additional details on the study design, see Bayliss et al , ). Patients with AL amyloidosis and cardiac dysfunction ( N = 108) completed an online survey including two measures of HRQoL: a generic measure [Short Form 36v2 (SF‐36v2 ® ) Health Survey (SF‐36v2); Maruish, ] and disease‐specific measure [Kansas City Cardiomyopathy Short Form (KCCQ‐12); Green et al , ].…”