Health-Related Quality of Life for Patients With Transthyretin Amyloid Cardiomyopathy: A Real-World Study in Europe, Canada, and Japan
Author(s)
Teresa Kauf, PhD1, Jack Wright, MSc2, Brianne Kerr, BSc2, Sam Williamson, BSc2, Mark Oellerich, MD3.
1Alnylam Pharmaceuticals, Cambridge, MA, USA, 2Adelphi Real World, Bollington, United Kingdom, 3Alnylam Switzerland GMBH, Zug, Switzerland.
1Alnylam Pharmaceuticals, Cambridge, MA, USA, 2Adelphi Real World, Bollington, United Kingdom, 3Alnylam Switzerland GMBH, Zug, Switzerland.
OBJECTIVES: Describe health-related quality of life (HRQoL) across New York Heart Association (NYHA) classes for transthyretin amyloidosis cardiomyopathy (ATTR-CM) patients in a real-world setting, as reported by patients.
METHODS: Secondary analyses using data from Adelphi ATTR Disease Specific ProgrammeTM, a cross-sectional survey of physicians and patients in Europe (France, Germany, Italy, Portugal, Spain, UK), Canada and Japan, September 2024-February 2025. Physicians reported patient demographics and NYHA class. Patients reported EQ-5D-5L, EQ-VAS and Kansas City Cardiomyopathy Question (KCCQ) outcomes. Patients were grouped by NYHA class at survey as I, II, and III. Analyses were descriptive.
RESULTS: Overall, 68 physicians provided data for 235 ATTR-CM patients with self-reported data. Mean (SD) patient age was 70.1 (11.6) years, 77.4% were male and 6.4% had a mixed cardiomyopathy-neuropathy phenotype. At survey, 16.6% (n=39) of patients were NYHA class I, 66.8% (n=157) II, 15.3% (n=36) III and 1.3% (n=3) IV. Patients reported a mean (SD) EQ-5D health utility of 0.82 (0.17) (I 0.91 [0.09], II 0.82 [0.18], III 0.76 [0.14]) and EQ-VAS score of 63.52 (18.03) (I 73.03 [16.65], II 64.17 [16.88], III 53.06 [15.50]). Mean (SD) KCCQ overall summary score was 63.80 (18.11) (I 74.01 [19.10], II 64.61 [17.69], III 51.63 [10.42]), and quality-of-life (QoL) score was 59.52 (19.34) (I 66.92 [19.71], II 60.88 [18.68], III 46.69 [16.60]). Patients reported that they had been bothered by symptoms of fatigue (88.5%), and shortness of breath (84.4%), and swelling in legs/feet (66.1%), over the prior 2 weeks.
CONCLUSIONS: ATTR-CM had a notable impact on HRQoL. Average scores for the EQ-5D and KCCQ were numerically lower among more severe NYHA classes. Symptoms of fatigue and shortness of breath were bothersome for most patients. The poor QoL outcomes reported at later stages of disease highlight the importance of early diagnosis, and timely initiation of treatments to maintain stability and slow progression.
METHODS: Secondary analyses using data from Adelphi ATTR Disease Specific ProgrammeTM, a cross-sectional survey of physicians and patients in Europe (France, Germany, Italy, Portugal, Spain, UK), Canada and Japan, September 2024-February 2025. Physicians reported patient demographics and NYHA class. Patients reported EQ-5D-5L, EQ-VAS and Kansas City Cardiomyopathy Question (KCCQ) outcomes. Patients were grouped by NYHA class at survey as I, II, and III. Analyses were descriptive.
RESULTS: Overall, 68 physicians provided data for 235 ATTR-CM patients with self-reported data. Mean (SD) patient age was 70.1 (11.6) years, 77.4% were male and 6.4% had a mixed cardiomyopathy-neuropathy phenotype. At survey, 16.6% (n=39) of patients were NYHA class I, 66.8% (n=157) II, 15.3% (n=36) III and 1.3% (n=3) IV. Patients reported a mean (SD) EQ-5D health utility of 0.82 (0.17) (I 0.91 [0.09], II 0.82 [0.18], III 0.76 [0.14]) and EQ-VAS score of 63.52 (18.03) (I 73.03 [16.65], II 64.17 [16.88], III 53.06 [15.50]). Mean (SD) KCCQ overall summary score was 63.80 (18.11) (I 74.01 [19.10], II 64.61 [17.69], III 51.63 [10.42]), and quality-of-life (QoL) score was 59.52 (19.34) (I 66.92 [19.71], II 60.88 [18.68], III 46.69 [16.60]). Patients reported that they had been bothered by symptoms of fatigue (88.5%), and shortness of breath (84.4%), and swelling in legs/feet (66.1%), over the prior 2 weeks.
CONCLUSIONS: ATTR-CM had a notable impact on HRQoL. Average scores for the EQ-5D and KCCQ were numerically lower among more severe NYHA classes. Symptoms of fatigue and shortness of breath were bothersome for most patients. The poor QoL outcomes reported at later stages of disease highlight the importance of early diagnosis, and timely initiation of treatments to maintain stability and slow progression.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR115
Topic
Patient-Centered Research
Topic Subcategory
Health State Utilities, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory), Rare & Orphan Diseases