BURDEN OF SYMPTOMS IN TRANSTHYRETIN AMYLOID CARDIOMYOPATHY: A UNITED STATES PHYSICIAN SURVEY DESCRIBING GENDER PERSPECTIVES
Author(s)
Janice Chung, MPH1, Ann Willet, PharmD1, Jade Garratt-Wheeldon, BSc2, Cynthia Khanji, PhD3, Feng Dai, PhD1, Alisha Braid, BSc2, Jack Wright, MSc2;
1Pfizer Inc., New York, NY, USA, 2Adelphi Real World, London, United Kingdom, 3Pfizer Canada, Lorraine, QC, Canada
1Pfizer Inc., New York, NY, USA, 2Adelphi Real World, London, United Kingdom, 3Pfizer Canada, Lorraine, QC, Canada
OBJECTIVES: To identify the most burdensome symptoms of transthyretin amyloid cardiomyopathy (ATTR-CM) from the perspectives of physicians in the United States (US).
METHODS: Secondary analyses were conducted using data from the Adelphi Real World ATTR Disease Specific Programme™, a cross-sectional survey of patients and physicians from August 2024 to February 2025 in the US. Physicians completed electronic record forms for up to 10 consecutively consulting patients with ATTR-CM, reporting patient demographics and clinical characteristics, and the top 10 most burdensome symptoms. Results were reported by gender.
RESULTS: Data for 256 patients were included, with a mean age of 63.9 ± 13.2 years; 156 (60.9%) were male and 100 (39.1%) females, with 55.9% identifying as white. At the time of the survey, patients were classified as New York Heart Association class I (21.9%), class II (54.3%), class III (19.9%), and class IV (3.9%). The top three symptoms, predominantly cardiac, were dyspnea/shortness of breath (39.1% overall; 44.2% male, 31.0% female), fatigue (26.2% overall; 28.8% male, 22.0% female), and edema (21.9% overall; 23.1% male, 20.0% female). Atrial fibrillation was also among the top five symptoms (16.8% overall; 15.4% male, 19.0% female).
CONCLUSIONS: The most burdensome symptoms for patients with ATTR-CM, as reported by US physicians, were similar across genders and commonly observed in cardiovascular diseases. Effective treatments for ATTR-CM should target these symptoms alongside addressing the underlying disease causes.
METHODS: Secondary analyses were conducted using data from the Adelphi Real World ATTR Disease Specific Programme™, a cross-sectional survey of patients and physicians from August 2024 to February 2025 in the US. Physicians completed electronic record forms for up to 10 consecutively consulting patients with ATTR-CM, reporting patient demographics and clinical characteristics, and the top 10 most burdensome symptoms. Results were reported by gender.
RESULTS: Data for 256 patients were included, with a mean age of 63.9 ± 13.2 years; 156 (60.9%) were male and 100 (39.1%) females, with 55.9% identifying as white. At the time of the survey, patients were classified as New York Heart Association class I (21.9%), class II (54.3%), class III (19.9%), and class IV (3.9%). The top three symptoms, predominantly cardiac, were dyspnea/shortness of breath (39.1% overall; 44.2% male, 31.0% female), fatigue (26.2% overall; 28.8% male, 22.0% female), and edema (21.9% overall; 23.1% male, 20.0% female). Atrial fibrillation was also among the top five symptoms (16.8% overall; 15.4% male, 19.0% female).
CONCLUSIONS: The most burdensome symptoms for patients with ATTR-CM, as reported by US physicians, were similar across genders and commonly observed in cardiovascular diseases. Effective treatments for ATTR-CM should target these symptoms alongside addressing the underlying disease causes.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HSD16
Topic
Health Service Delivery & Process of Care
Disease
SDC: Cardiovascular Disorders (including MI, Stroke, Circulatory), SDC: Rare & Orphan Diseases