A Real-World Study on Healthcare Resource Utilisation and Patient Care for People With Transthyretin Amyloid Polyneuropathy
Author(s)
Gengshi Chen, MSc1, Klas Rikner, PhD2, Susan Grandy, PhD3, Eric T. Wittbrodt, MPH3, Joanna Huang, MS3, Jack Wright, MSc4, Sam Williamson, BSc4.
1AstraZeneca, Cambridge, United Kingdom, 2AstraZeneca, Gothenburg, Sweden, 3AstraZeneca, Wilmington, DE, USA, 4Adelphi Real World, Bollington, United Kingdom.
1AstraZeneca, Cambridge, United Kingdom, 2AstraZeneca, Gothenburg, Sweden, 3AstraZeneca, Wilmington, DE, USA, 4Adelphi Real World, Bollington, United Kingdom.
OBJECTIVES: Describe patient care and healthcare resource utilization (HCRU) for people with transthyretin amyloid polyneuropathy (ATTR-PN), using real-world interim data.
METHODS: Interim analysis of data from the Adelphi ATTR Disease Specific Programme™, a cross-sectional survey in Europe (France, Germany, Italy, the United Kingdom [UK]) and the United States (US), October-December 2024. Physicians reported patient demographics, management and HCRU for their next consulting patients. Analyses were descriptive.
RESULTS: Overall, 75 physicians provided data for 265 patients, 142 in Europe and 123 in the US. Of these, 70.2% had ATTR-PN and 29.8% had a mixed cardiomyopathy-neuropathy phenotype. Mean (standard deviation; SD) age was 55.3 (15.2) years, 64.2% were male, and 82.0% were White. Median (interquartile range) disease duration was 2.0 (1.0-3.4) years.Diagnosing physicians included neurologists (59.4%), cardiologists (15.7%), and neuromuscular specialists (6.5%). Patients had a mean (SD) 9.4 (27.4) consultations during the 12 months prior to survey, most commonly neurologists (77.7%), cardiologists (41.9%), and/or primary care physicians (35.8%). ATTR diagnosis required a mean (SD) 11.0 (6.7) assessments, including electromyography (75.6%), nerve conduction (71.0%), and electrocardiogram (59.9%), and 6.5 (4.9) types of assessments were performed to monitor ATTR. In the 12 months prior to survey, 14.6% of patients were hospitalized due to ATTR, of which 64.5% of patients were admitted through the emergency room. Overall, mobility aids were required by 47.2%, typically a cane/walking stick (33.6% of all patients). 54.0% of patients had a caregiver, of which 80.1% were their partner/spouse. Caregivers provided an estimated mean (SD) of 12.9 (11.5) hours of weekly care, with activities such as transportation (63.8%) and housecleaning (62.2%).
CONCLUSIONS: ATTR-PN diagnosis and management required multi-disciplinary care and ongoing assessments to monitor disease progression. Approximately half of patients required mobility aids and/or caregiver support. Findings from this study confirm the high burden of ATTR-PN for both patients and caregiver alike.
METHODS: Interim analysis of data from the Adelphi ATTR Disease Specific Programme™, a cross-sectional survey in Europe (France, Germany, Italy, the United Kingdom [UK]) and the United States (US), October-December 2024. Physicians reported patient demographics, management and HCRU for their next consulting patients. Analyses were descriptive.
RESULTS: Overall, 75 physicians provided data for 265 patients, 142 in Europe and 123 in the US. Of these, 70.2% had ATTR-PN and 29.8% had a mixed cardiomyopathy-neuropathy phenotype. Mean (standard deviation; SD) age was 55.3 (15.2) years, 64.2% were male, and 82.0% were White. Median (interquartile range) disease duration was 2.0 (1.0-3.4) years.Diagnosing physicians included neurologists (59.4%), cardiologists (15.7%), and neuromuscular specialists (6.5%). Patients had a mean (SD) 9.4 (27.4) consultations during the 12 months prior to survey, most commonly neurologists (77.7%), cardiologists (41.9%), and/or primary care physicians (35.8%). ATTR diagnosis required a mean (SD) 11.0 (6.7) assessments, including electromyography (75.6%), nerve conduction (71.0%), and electrocardiogram (59.9%), and 6.5 (4.9) types of assessments were performed to monitor ATTR. In the 12 months prior to survey, 14.6% of patients were hospitalized due to ATTR, of which 64.5% of patients were admitted through the emergency room. Overall, mobility aids were required by 47.2%, typically a cane/walking stick (33.6% of all patients). 54.0% of patients had a caregiver, of which 80.1% were their partner/spouse. Caregivers provided an estimated mean (SD) of 12.9 (11.5) hours of weekly care, with activities such as transportation (63.8%) and housecleaning (62.2%).
CONCLUSIONS: ATTR-PN diagnosis and management required multi-disciplinary care and ongoing assessments to monitor disease progression. Approximately half of patients required mobility aids and/or caregiver support. Findings from this study confirm the high burden of ATTR-PN for both patients and caregiver alike.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HSD76
Topic
Health Service Delivery & Process of Care
Disease
SDC: Neurological Disorders, SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)