OBJECTIVES : Hereditary transthyretin amyloidosis (hATTR) is an ultra-rare and fatal disease with high unmet need. This study explored hATTR caregiver burden in terms of health-related quality of life (HRQoL) and productivity.

METHODS : A cross-sectional online survey, including Hospital Anxiety and Depression Scale (HADS) and caregiver and patient characteristics questions, was developed to assess hATTR caregiver burden. The survey was reviewed and piloted by Amyloidosis Research Consortium (ARC) UK. A companion general population survey collected HADS and chronic health conditions data. Caregivers were recruited via a patient panel and ARC UK, in the UK, US, Canada, Australia and New Zealand. A matched general population sample was recruited in the UK via a healthcare panel agency.

RESULTS : Thirty-six caregivers and matched controls completed the survey (n=72). Caregivers had a mean age of 56 years (SD=12.5), were pre-dominantly female (81%) and cared for their partner/spouse (78%), and over half (58%) were not employed. The disease severity of the people they cared for was varied: one third (33%) required no assistance walking, over half (58%) required various levels of assistance walking and 9% required a wheelchair/were bedridden. On average, caregivers spent 5.8 hours (SD=6.2) daily on practical care and 4.1 hours (SD=6.1) daily on emotional support; over half (56%) indicated that they had changed their employment (stopped work, reduced hours, changed jobs) due to providing hATTR care. Caregivers had significantly higher HADS anxiety (M=9.3 vs. M=6.1, p <0.01) and depression (M=7.6 vs. M=4.4, p <0.01) scores than the matched general population. Furthermore, caregivers were more likely to report sleep problems (33% vs. 8%, p <0.01) and stress (42% vs. 0%, p <0.001) as chronic conditions than controls.

CONCLUSIONS : The results of this study indicate that caring for a person with hATTR can have a considerable negative impact on caregivers HRQoL and productivity.