Abstract 4341180: Describing Burden For Transthyretin Amyloid Cardiomyopathy Patients And Their Caregivers In The United States: A Real-World Survey

Abstract

Background: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare form of heart failure. Symptoms can be burdensome and management often involves caregiver support. However, real-world data describing burden in ATTR-CM are limited. Objective: To describe the burden of ATTR-CM among patients and their caregivers in the United States (US). Methods: Secondary analyses of data from the Adelphi Real World ATTR Disease Specific Programme™, a cross-sectional survey of ATTR-treating physicians and their patients in the US from August 2024 – February 2025. Physicians reported patient demographics and caregiver support. Patients and their accompanying non-professional caregivers were invited to report disease burden, including the Work Productivity and Activity Impairment questionnaire (WPAI), and the Zarit Burden Interview (ZBI) for caregivers. Analyses were descriptive. Results: Overall, 33 physicians provided data for 256 patients – of whom 58 self-reported data – and 15 caregivers provided data. Mean (standard deviation; SD) patient age was 63.9 (13.2) years and 60.9% of patients were male. At survey, 21.9% of patients were New York Heart Association (NYHA) class I, 54.3% class II, 19.9% class III, and 3.9% class IV. Median (interquartile range; IQR) disease duration was 1.9 (1.3–3.0) years. Overall, 42.2% of patients had a caregiver, commonly their partner/spouse (61.7%), their child (21.0%), and/or a professional caregiver (16.0%). Caregiver support was often required with housecleaning/home maintenance (68.0%), transportation (65.3%), and shopping/meal preparation (64.0%). Patients cited fear about symptoms getting worse (94.6%) and loss of independence (87.9%) as negatively impacting quality of life (QoL). For the WPAI ( Figure 1 ), patients reported a mean (SD) percent work time missed of 12.5% (19.2%). For the WPAI ( Figure 2 ), caregivers reported a mean (SD) percent work time missed of 53.8% (50.0%). For the ZBI, caregivers reported a mean (SD) score of 39.1 (20.1). Conclusion: Despite an average disease duration of >2 years, over two fifths of patients required caregiver support. Most patients reported loss of independence as negatively impacting their QoL, with work and activity impairment evident in both patient and caregiver WPAI data. This substantial burden on patients and their caregivers underscores the need for interventions to address clinical outcomes and reduce caregiver dependence.