Describing the Burden of Transthyretin Amyloid Cardiomyopathy (ATTR-CM) on Patients and Their Caregivers: A Real-World Survey in Europe, the United States, and Japan
Author(s)
Sanjana Chandrasekar, MPH1, Jack Wright, MSc2, Jade Garratt-Wheeldon, BSc2, Janice Chung, MPH1, Mónica S. Inês, MSc, PhD3, Christine L. Baker, MBA MPH JD1, Ann Willet, MBA PharmD1, Pedro Ferreira Branquinho, Postgrad3, Feng Dai, PhD1, Sam Williamson, BSc2, Katherine Smethers, BSc2.
1Pfizer, New York, NY, USA, 2Adelphi Real World, Bollington, United Kingdom, 3Pfizer, Porto Salvo, Portugal.
1Pfizer, New York, NY, USA, 2Adelphi Real World, Bollington, United Kingdom, 3Pfizer, Porto Salvo, Portugal.
OBJECTIVES: Describe disease burden among transthyretin amyloid cardiomyopathy (ATTR-CM) patients and their caregivers in a real-world setting.
METHODS: Descriptive analyses of data from the Adelphi ATTR Disease Specific ProgrammeTM, a cross-sectional survey of physicians and their patients in Europe (France, Germany, Italy, Spain, and the UK), the US, and Japan, from August 2024-February 2025. Physicians reported patient demographics and caregiver support. Patients and caregivers voluntarily reported aspects of disease burden, including the Work Productivity and Activity Impairment questionnaire (WPAI) regarding the prior 7 days, and Zarit Burden Interview (ZBI) with scores from 0-88.
RESULTS: Overall, 226 physicians provided data for 1006 patients (n=630 Europe, n=256 US, n=120 Japan), 279 patients and 121 caregivers self-reported data. Mean (SD) patient age was 70.4 (12.8) years, 70% were male. Mean (SD) disease duration was 2.9 (2.8) years. At survey, 19% of patients were New York Heart Association class I, 61% class II, 18% class III and 2% class IV. Overall, 58% of patients required caregiver assistance; frequently from their partner/spouse (68%). Physicians reported patients required help with home maintenance (59%), transportation (59%), and shopping/meal preparation (56%). Loss of independence was reported by 85% of patients as negatively impacting their quality of life (QoL). Mean (SD) ZBI score reported by caregivers was 29.4 (17.3). Per WPAI, patients reported mean (SD) % total work impairment of 34.0 (26.2), activity impairment 43.0 (24.7), and work time missed 11.1 (20.6). Caregivers reported % total work impairment of 33.1 (29.0), activity impairment 37.7 (25.3), and work time missed 17.9 (35.5).
CONCLUSIONS: Loss of independence negatively impacted the QoL of nearly all ATTR-CM patients surveyed. Over half required caregiver support. Both patients and caregivers reported notable impairment of their work and living. These results highlight the burden associated with this condition, and the urgent need for timely interventions to support those affected.
METHODS: Descriptive analyses of data from the Adelphi ATTR Disease Specific ProgrammeTM, a cross-sectional survey of physicians and their patients in Europe (France, Germany, Italy, Spain, and the UK), the US, and Japan, from August 2024-February 2025. Physicians reported patient demographics and caregiver support. Patients and caregivers voluntarily reported aspects of disease burden, including the Work Productivity and Activity Impairment questionnaire (WPAI) regarding the prior 7 days, and Zarit Burden Interview (ZBI) with scores from 0-88.
RESULTS: Overall, 226 physicians provided data for 1006 patients (n=630 Europe, n=256 US, n=120 Japan), 279 patients and 121 caregivers self-reported data. Mean (SD) patient age was 70.4 (12.8) years, 70% were male. Mean (SD) disease duration was 2.9 (2.8) years. At survey, 19% of patients were New York Heart Association class I, 61% class II, 18% class III and 2% class IV. Overall, 58% of patients required caregiver assistance; frequently from their partner/spouse (68%). Physicians reported patients required help with home maintenance (59%), transportation (59%), and shopping/meal preparation (56%). Loss of independence was reported by 85% of patients as negatively impacting their quality of life (QoL). Mean (SD) ZBI score reported by caregivers was 29.4 (17.3). Per WPAI, patients reported mean (SD) % total work impairment of 34.0 (26.2), activity impairment 43.0 (24.7), and work time missed 11.1 (20.6). Caregivers reported % total work impairment of 33.1 (29.0), activity impairment 37.7 (25.3), and work time missed 17.9 (35.5).
CONCLUSIONS: Loss of independence negatively impacted the QoL of nearly all ATTR-CM patients surveyed. Over half required caregiver support. Both patients and caregivers reported notable impairment of their work and living. These results highlight the burden associated with this condition, and the urgent need for timely interventions to support those affected.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR57
Topic
Economic Evaluation, Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory), Rare & Orphan Diseases