Disability Resource Utilization and Caregiver Support Among a Real-World Sample of Patients With Transthyretin Amyloidosis With Polyneuropathy
Author(s)
Gengshi Chen, MSc1, Klas Rikner, PhD2, Susan Grandy, PhD MBA3, Jack Wright, MSc4, Jade Garratt-Wheeldon, BSc4, Alisha Braid, BSc4.
1Global Market Access and Pricing, Health Economics and Payer Evidence, BioPharmaceuticals, AstraZeneca, Cambridge, United Kingdom, 2Global Market Access and Pricing, Health Economics and Payer Evidence, BioPharmaceuticals, AstraZeneca, Gothenburg, Sweden, 3Global Market Access and Pricing, BioPharmaceuticals, AstraZeneca, Wilmington, DE, USA, 4Adelphi Real World, Bollington, United Kingdom.
1Global Market Access and Pricing, Health Economics and Payer Evidence, BioPharmaceuticals, AstraZeneca, Cambridge, United Kingdom, 2Global Market Access and Pricing, Health Economics and Payer Evidence, BioPharmaceuticals, AstraZeneca, Gothenburg, Sweden, 3Global Market Access and Pricing, BioPharmaceuticals, AstraZeneca, Wilmington, DE, USA, 4Adelphi Real World, Bollington, United Kingdom.
OBJECTIVES: Describe resource utilisation, caregiver support and healthcare consultations relating to physical function among patients with transthyretin amyloidosis with polyneuropathy (ATTR-PN), across different polyneuropathy disability (PND) scores.
METHODS: Analysis of data from the Adelphi ATTR Disease Specific Programme™, a cross-sectional survey conducted in Brazil, China, Europe (France, Germany, Italy, the United Kingdom [UK]), and the United States (US), October 2024-May 2025. Physicians reported patient demographics and clinical characteristics (including PND scores), management, and HCRU for consecutively consulting patients. Analyses were descriptive.
RESULTS: Overall, 245 physicians reported data for 1,179 patients (PND 0: 104, I: 413, II: 366, IIIA: 184, IIIB: 82, IV: 30), of whom 58.3% had polyneuropathy only and 41.7% a mixed cardiomyopathy-polyneuropathy phenotype. Mean (SD) age was 58.2 (15.7), and 65.5% were male. Median (IQR) disease duration was 3.1 (1.7-5.3) years. Overall, 43.9% of patients utilised a mobility aid (PND 0-IV: 20.2%-93.3%), and 45.4% had modified their home (PND 0-IV: 25.9%-72.7%), due to their condition. Most patients (60.9%) required additional support from a caregiver (PND 0-IV: 36.1%-96.3%). Among those with a caregiver, activities that frequently required additional support included home maintenance (64.9%), transportation (64.4%), and shopping and meal preparation (63.0%). One quarter (25.1%) of patients had consulted with a physical therapist in the 12 months prior to survey (PND 0-IV: 17.3%-48.3%), and 8.4% with either an occupational therapist (4.8%-20.7%), or rehabilitation medicine specialist (6.7%-20.7%).
CONCLUSIONS: Results from this study highlight the levels of disability and impairment experienced by patients with ATTR-PN. Most patients required caregiver support with their activities of daily living, and many had modified their homes to better accommodate their condition, potentially resulting in significant direct and indirect costs. Earlier detection and treatment of ATTR-PN may help to mitigate the burden on both patients and caregivers.
METHODS: Analysis of data from the Adelphi ATTR Disease Specific Programme™, a cross-sectional survey conducted in Brazil, China, Europe (France, Germany, Italy, the United Kingdom [UK]), and the United States (US), October 2024-May 2025. Physicians reported patient demographics and clinical characteristics (including PND scores), management, and HCRU for consecutively consulting patients. Analyses were descriptive.
RESULTS: Overall, 245 physicians reported data for 1,179 patients (PND 0: 104, I: 413, II: 366, IIIA: 184, IIIB: 82, IV: 30), of whom 58.3% had polyneuropathy only and 41.7% a mixed cardiomyopathy-polyneuropathy phenotype. Mean (SD) age was 58.2 (15.7), and 65.5% were male. Median (IQR) disease duration was 3.1 (1.7-5.3) years. Overall, 43.9% of patients utilised a mobility aid (PND 0-IV: 20.2%-93.3%), and 45.4% had modified their home (PND 0-IV: 25.9%-72.7%), due to their condition. Most patients (60.9%) required additional support from a caregiver (PND 0-IV: 36.1%-96.3%). Among those with a caregiver, activities that frequently required additional support included home maintenance (64.9%), transportation (64.4%), and shopping and meal preparation (63.0%). One quarter (25.1%) of patients had consulted with a physical therapist in the 12 months prior to survey (PND 0-IV: 17.3%-48.3%), and 8.4% with either an occupational therapist (4.8%-20.7%), or rehabilitation medicine specialist (6.7%-20.7%).
CONCLUSIONS: Results from this study highlight the levels of disability and impairment experienced by patients with ATTR-PN. Most patients required caregiver support with their activities of daily living, and many had modified their homes to better accommodate their condition, potentially resulting in significant direct and indirect costs. Earlier detection and treatment of ATTR-PN may help to mitigate the burden on both patients and caregivers.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HSD35
Topic
Economic Evaluation, Health Service Delivery & Process of Care
Disease
Neurological Disorders, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)