OBJECTIVES: To assess the burden of transthyretin (ATTR) amyloidosis and disease-related ambulatory disability on patients’ health-related quality of life (HRQOL) and to compare the HRQOL profile of patients from different study samples.

METHODS: Baseline SF-36v2® Health Survey (SF-36v2) data were collected from 83 adults with ATTR amyloidosis enrolled in a longitudinal online observational study and 172 adults enrolled in a randomized clinical trial (ClinicalTrials.gov ID: NCT01737398). Mean SF-36v2 scores from both patient samples were compared with scores from a United States general population (USGP) sample (N=4036). Regression models were used to adjust the patient samples to match the age and gender distribution of the USGP sample, and univariate analysis of variance models were used to compare mean SF-36v2 scores. Similar approaches were conducted within subgroups with varying ambulatory disability. Results were interpreted using effect sizes (Cohen’s d) and minimally important difference (MID) thresholds established for the SF-36v2 subscale and summary scores.

RESULTS: Patients with ATTR amyloidosis from the observational study scored worse on most domains of the SF-36v2, compared with the USGP, by more than an MID: physical functioning, role-physical, general health, vitality, social functioning, role-emotional, and the physical component summary score (ps<0.001), with the greatest burden in general health (d=0.48). Patients from the clinical trial showed a similar HRQOL profile to those in the observational study, with greater deficits in physical functioning and role-physical (ps<0.03). Patients from the observational study who reported ambulatory disability exhibited substantial burden on all domains of the SF-36v2, except mental health, when compared to the USGP (ps<0.02, ds>0.43); differences between the two patient samples with ambulatory disability were not statistically significant.

CONCLUSIONS: Patients with ATTR amyloidosis, regardless of data source, experience clinically meaningful deficits in multiple areas of HRQOL relative to the USGP, with greater HRQOL burden among those patients who need mobility assistance.