Characterizing the High Disease Burden of Transthyretin Amyloidosis for Patients and Caregivers

Michelle Stewart, Shannon Shaffer, Brian Murphy, Jane Loftus, Jose Alvir, Michael Cicchetti, William R Lenderking, Michelle Stewart, Shannon Shaffer, Brian Murphy, Jane Loftus, Jose Alvir, Michael Cicchetti, William R Lenderking

Abstract

Introduction: Transthyretin amyloidosis (ATTR amyloidosis), whether manifesting as familial amyloid polyneuropathy (ATTR-PN) or cardiomyopathy (ATTR-CM), is a progressive, debilitating, and often fatal, rare disease requiring significant caregiver support. This study aims to better characterize the burden of disease for ATTR amyloidosis patients and caregivers.

Methods: Patients and caregivers in the USA and Spain were recruited through patient advocacy groups to complete a cross-sectional survey. Assessments included the 12-Item Short Form Health Survey, the Work Productivity and Activity Impairment Questionnaire, the Zarit Burden Interview, pain and symptom measures, health care resource use measures, and caregiving burden measures.

Results: Respondents included 60 ATTR amyloidosis patients and 32 caregivers. Patients registered scores up to two standard deviations below normal for physical health, impairment in quality of life, and reduced work productivity. Patients with liver transplant versus without liver transplant reported better overall outcomes, and those without liver transplant reported a greater impact on physical versus mental health. A high rate of health care utilization in the 3 months prior to the survey was reported by patients. Caregivers reported substantial burden, including poor mental health, work impairment, and much time spent providing care (mean 45.9 h/week). Work productivity was impacted for employed patients and caregivers.

Conclusion: ATTR amyloidosis was associated with high levels of impairment in many domains, including physical health, quality of life, and reduced productivity. Providing care for ATTR amyloidosis patients is associated with a negative impact on the mental health of caregivers. These results highlight the substantial burden of ATTR amyloidosis for patients and caregivers.

Trial registration: ClinicalTrials.gov: NCT01604122.

Funding: Pfizer.

Keywords: Burden of disease; Caregivers; Familial amyloid polyneuropathy; Transthyretin amyloidosis; Transthyretin cardiomyopathy.

Figures

Fig. 1
Fig. 1
Patient health. EQ-5D-3L EuroQol–5 Dimensions–3 Levels questionnaire, EQ-VAS EuroQol visual analog scale, SD standard deviation
Fig. 2
Fig. 2
Patient work productivity by country and transplant status. SD standard deviation
Fig. 3
Fig. 3
Patient quality of life. SD standard deviation
Fig. 4
Fig. 4
Caregiver burden. SD standard deviation
Fig. 5
Fig. 5
Caregiver health. SD standard deviation
Fig. 6
Fig. 6
Caregiver work productivity. SD standard deviation, WPAI-SH Work Productivity and Activity Impairment Specific Health questionnaire

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Source: PubMed

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