TREATMENT PATTERNS AND HEALTH-RELATED QUALITY OF LIFE ACROSS DISEASE STAGES AMONG A REAL-WORLD SAMPLE OF PATIENTS WITH MIXED PHENOTYPE TRANSTHYRETIN AMYLOIDOSIS
Author(s)
Gengshi Chen, MSc1, Klas Rikner, PhD2, Mia Papas, PhD3, Susan Grandy, PhD4, Jack Wright, MSc5, Jade Garratt-Wheeldon, PGCert5, Brianne Kerr, BSc5;
1AstraZeneca, Global Market Access and Pricing, Health Economics and Payer Evidence, BioPharmaceuticals, Cambridge, United Kingdom, 2AstraZeneca, Global Market Access and Pricing, Health Economics and Payer Evidence, BioPharmaceuticals, Gothenburg, Sweden, 3AstraZeneca, US Evidence, Real World Evidence, BioPharmaceuticals, Wilmington, DE, USA, 4AstraZeneca, Global Market Access and Pricing, BioPharmaceuticals, Wilmington, DE, USA, 5Adelphi Real World, Bollington, United Kingdom
1AstraZeneca, Global Market Access and Pricing, Health Economics and Payer Evidence, BioPharmaceuticals, Cambridge, United Kingdom, 2AstraZeneca, Global Market Access and Pricing, Health Economics and Payer Evidence, BioPharmaceuticals, Gothenburg, Sweden, 3AstraZeneca, US Evidence, Real World Evidence, BioPharmaceuticals, Wilmington, DE, USA, 4AstraZeneca, Global Market Access and Pricing, BioPharmaceuticals, Wilmington, DE, USA, 5Adelphi Real World, Bollington, United Kingdom
OBJECTIVES: Describe treatment patterns and health-related quality of life (HRQoL), among patients with mixed phenotype transthyretin (ATTR) amyloidosis, across polyneuropathy disability (PND) and New York Heart Association (NYHA) scores.
METHODS: Descriptive analysis of secondary data from the Adelphi ATTR Disease Specific Programme™, a cross-sectional survey in France, Germany, Italy, Spain, the UK, the US, China, and Brazil (October 2024-May 2025). Physicians reported sociodemographics, treatment history, NYHA classes, and PND scores. Patients reported HRQoL via the Norfolk QoL-DN and EQ-5D-5L (German tariff).
RESULTS: Overall, 159 physicians provided data for 490 patients with mixed phenotype. Mean (SD) age was 65.0 (14.7) years and 71.6% were male. At survey, 10.6% were PND 0, 30.8% I, 32.9% II, 15.5% IIIA, 6.1% IIIB and 4.1% IV, and 19.4% were NYHA I, 59.0% were II, 18.0% were III, and 3.7% were IV. At survey, 77.1% of patients were prescribed disease modifying therapies (PND 0 61.5%, I 82.1%, II 82.0%, IIIA 71.1%, IIIB 80.0%, IV 60.0%; NYHA I 75.8%, II 81.0%, III 68.2%, IV 66.7%). Physicians were not completely satisfied with treatment for 36.2% of patients (ranging from 24.2-83.6% at PND 0-IV and 21.9-69.2% at NYHA I-IV). For patients with Norfolk QoL-DN data (n=112), mean (SD) total score ranged from 21.6 (14.3) at PND I to 89.0 (29.0) at IV, and 25.7 (20.8) at NYHA I to 82.2 (30.9) at IV. Mean (SD) EQ-5D-5L scores (n=120) ranged from 0.92 (0.11) at PND 0 to 0.38 (0.29) at IV, and 0.87 (0.13) at NYHA I to 0.51 (0.33) at IV.
CONCLUSIONS: Results from this real-world study indicate the negative impacts of ATTR amyloidosis on the HRQoL of patients with mixed phenotype across all disease stages. Despite treatment, many patients reported poor disease-specific and generic HRQoL. These findings underscore the need for continued treatment development and early intervention to help maintain patient HRQoL.
METHODS: Descriptive analysis of secondary data from the Adelphi ATTR Disease Specific Programme™, a cross-sectional survey in France, Germany, Italy, Spain, the UK, the US, China, and Brazil (October 2024-May 2025). Physicians reported sociodemographics, treatment history, NYHA classes, and PND scores. Patients reported HRQoL via the Norfolk QoL-DN and EQ-5D-5L (German tariff).
RESULTS: Overall, 159 physicians provided data for 490 patients with mixed phenotype. Mean (SD) age was 65.0 (14.7) years and 71.6% were male. At survey, 10.6% were PND 0, 30.8% I, 32.9% II, 15.5% IIIA, 6.1% IIIB and 4.1% IV, and 19.4% were NYHA I, 59.0% were II, 18.0% were III, and 3.7% were IV. At survey, 77.1% of patients were prescribed disease modifying therapies (PND 0 61.5%, I 82.1%, II 82.0%, IIIA 71.1%, IIIB 80.0%, IV 60.0%; NYHA I 75.8%, II 81.0%, III 68.2%, IV 66.7%). Physicians were not completely satisfied with treatment for 36.2% of patients (ranging from 24.2-83.6% at PND 0-IV and 21.9-69.2% at NYHA I-IV). For patients with Norfolk QoL-DN data (n=112), mean (SD) total score ranged from 21.6 (14.3) at PND I to 89.0 (29.0) at IV, and 25.7 (20.8) at NYHA I to 82.2 (30.9) at IV. Mean (SD) EQ-5D-5L scores (n=120) ranged from 0.92 (0.11) at PND 0 to 0.38 (0.29) at IV, and 0.87 (0.13) at NYHA I to 0.51 (0.33) at IV.
CONCLUSIONS: Results from this real-world study indicate the negative impacts of ATTR amyloidosis on the HRQoL of patients with mixed phenotype across all disease stages. Despite treatment, many patients reported poor disease-specific and generic HRQoL. These findings underscore the need for continued treatment development and early intervention to help maintain patient HRQoL.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR27
Topic
Patient-Centered Research
Topic Subcategory
Health State Utilities, Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Cardiovascular Disorders (including MI, Stroke, Circulatory), SDC: Neurological Disorders, SDC: Rare & Orphan Diseases