Burden of Disease and Healthcare Resource Use in Patients With Hereditary Transthyretin Amyloidosis in Spain: Findings From the OverTTuRe Global Study
Author(s)
Lucia Galan, MD1, Pablo García, MD2, Margarita Capel Sanchez, MSc3, Marta Castro Gasch, MSc3, Sergio Cedillo, MSc3, Alicia Eisman, MD4, Krister Järbrink, BSc, MPhil, PhD5, Joaquín Sánchez-Covisa Hernández, MSc4.
1Neurology Service, Hospital Universitario Clínico San Carlos, Madrid, Spain, 2Cardiology Service, Hospital Universitario Puerta de Hierro, Majadahonda, Spain, 3Corporate Affairs and Market Access Department, BioPharmaceuticals, AstraZeneca, Madrid, Spain, 4Medical and Regulatory Affairs Department, BioPharmaceuticals Medical, AstraZeneca, Madrid, Spain, 5Cardiovascular, Renal and Metabolism Evidence, BioPharmaceuticals Medical, AstraZeneca, Gothenburg, Sweden.
1Neurology Service, Hospital Universitario Clínico San Carlos, Madrid, Spain, 2Cardiology Service, Hospital Universitario Puerta de Hierro, Majadahonda, Spain, 3Corporate Affairs and Market Access Department, BioPharmaceuticals, AstraZeneca, Madrid, Spain, 4Medical and Regulatory Affairs Department, BioPharmaceuticals Medical, AstraZeneca, Madrid, Spain, 5Cardiovascular, Renal and Metabolism Evidence, BioPharmaceuticals Medical, AstraZeneca, Gothenburg, Sweden.
OBJECTIVES: Hereditary transthyretin (ATTRv) amyloidosis is a rare, progressive, life-threatening disease caused by variants resulting in amyloid accumulation in organs and tissues. It mainly presents with peripheral and autonomic neuropathy, but can also affect the heart, kidneys, and eyes. OverTTuRe is a multi-country study providing a comprehensive view of the disease’s natural history. This analysis presents data from Spain, focusing on healthcare resource use and mortality.
METHODS: Retrospective observational study of medical records from 11 Spanish hospitals. The Coutinho staging, used to assess polyneuropathy severity -ranging from 0 (presymptomatic) to 3 (inability to walk)- was collected.
RESULTS: 212 patients with ATTRv amyloidosis were included; 135 classified as Coutinho stage 1-2 (86.5% of the cohort with staging information) with the remainder Stage 0 (12.2%) and Stage 3 (1.3%).
Mean age at diagnosis was 60.9 years (SD=15.2); 60.8% were male; median time from first manifestation to diagnosis was 7.9 months (IQR=0.6-44.6).
Average follow-up was 57.9 (SD=38.4) months. During follow-up, 40.1% were hospitalized at least once with 0.24 (SD=0.57) hospitalizations PPPY, and mean stay of 10.0 (SD=18.8) days per hospitalization (for Coutinho stage 1-2 patients: 34.9%, 0.19 [SD=0.44] hospitalizations PPPY, and 11.7 [SD=24.5] days). In the same period, 58.9% visited the Emergency Room (ER), with a mean of 0.77 (SD=1.66) admissions PPPY (for Coutinho stage 1-2 patients: 57.9% and 0.54 [SD=1.05] admissions PPPY). Specialist visits occurred in 95.2% with a mean of 5.6 (SD=6.7) visits PPPY (for Coutinho stage 1-2 patients; 95.4% and 5.6 [SD=7.2] visits PPPY). Mortality was 14.4% (13.3% in Coutinho 1-2).
CONCLUSIONS: ATTRv amyloidosis imposes a substantial burden on patients and the healthcare system, as evidenced by high rates of hospitalizations, ER visits, specialist consultations, and mortality even in early stages. These findings underscore an urgent need for improved clinical management and initiation of disease-modifying therapies to mitigate the impact of this progressive condition.
METHODS: Retrospective observational study of medical records from 11 Spanish hospitals. The Coutinho staging, used to assess polyneuropathy severity -ranging from 0 (presymptomatic) to 3 (inability to walk)- was collected.
RESULTS: 212 patients with ATTRv amyloidosis were included; 135 classified as Coutinho stage 1-2 (86.5% of the cohort with staging information) with the remainder Stage 0 (12.2%) and Stage 3 (1.3%).
Mean age at diagnosis was 60.9 years (SD=15.2); 60.8% were male; median time from first manifestation to diagnosis was 7.9 months (IQR=0.6-44.6).
Average follow-up was 57.9 (SD=38.4) months. During follow-up, 40.1% were hospitalized at least once with 0.24 (SD=0.57) hospitalizations PPPY, and mean stay of 10.0 (SD=18.8) days per hospitalization (for Coutinho stage 1-2 patients: 34.9%, 0.19 [SD=0.44] hospitalizations PPPY, and 11.7 [SD=24.5] days). In the same period, 58.9% visited the Emergency Room (ER), with a mean of 0.77 (SD=1.66) admissions PPPY (for Coutinho stage 1-2 patients: 57.9% and 0.54 [SD=1.05] admissions PPPY). Specialist visits occurred in 95.2% with a mean of 5.6 (SD=6.7) visits PPPY (for Coutinho stage 1-2 patients; 95.4% and 5.6 [SD=7.2] visits PPPY). Mortality was 14.4% (13.3% in Coutinho 1-2).
CONCLUSIONS: ATTRv amyloidosis imposes a substantial burden on patients and the healthcare system, as evidenced by high rates of hospitalizations, ER visits, specialist consultations, and mortality even in early stages. These findings underscore an urgent need for improved clinical management and initiation of disease-modifying therapies to mitigate the impact of this progressive condition.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HSD16
Topic
Health Service Delivery & Process of Care, Study Approaches
Disease
Neurological Disorders, No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases