Hereditary Transthyretin Amyloidosis (ATTRv) Below the Age of 70 Healthcare Resource Utilization and Mortality
Author(s)
Jonas Wixner, MD, PhD1, Björn Pilebro, MD, PhD1, Gustav J. Smith, MD, PhD2, Katarina Johansson, MSc, PhD3, Marcus Thuresson, PhD4, Krister Järbrink, MPhil, PhD5, Klas Rikner, PhD6.
1Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden, 2The Wallenberg Laboratory, Gothenburg University, Gothenburg, Sweden, 3Cardiovascular, Renal and Metabolism, Medical Department, BioPharmaceuticals, AstraZeneca, Stockholm, Sweden, 4Statisticon AB, Uppsala, Sweden, 5Cardiovascular, Renal and Metabolism Evidence, BioPharmaceuticals Medical, AstraZeneca, Gothenburg, Sweden, 6Global Market Access and Pricing, Health Economics and Payer Evidence, BioPharmaceuticals, AstraZeneca, Gothenburg, Sweden.
1Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden, 2The Wallenberg Laboratory, Gothenburg University, Gothenburg, Sweden, 3Cardiovascular, Renal and Metabolism, Medical Department, BioPharmaceuticals, AstraZeneca, Stockholm, Sweden, 4Statisticon AB, Uppsala, Sweden, 5Cardiovascular, Renal and Metabolism Evidence, BioPharmaceuticals Medical, AstraZeneca, Gothenburg, Sweden, 6Global Market Access and Pricing, Health Economics and Payer Evidence, BioPharmaceuticals, AstraZeneca, Gothenburg, Sweden.
OBJECTIVES: Transthyretin (ATTR) amyloidosis is a clinically heterogeneous and fatal condition that may present in patients with acquired or hereditary forms of the disease. Diagnostic delay is common due to the non-specific nature of the initial symptoms, which often mimic more common conditions. This analysis, which is part of OverTTuRe, a global multi-country study, focuses on hereditary ATTR (ATTRv) amyloidosis and patients receiving the diagnosis before the age of 70.
METHODS: Data were generated by merging data from the Swedish national quality registry for ATTR amyloidosis (SveATTR) from January 2000 to November 2024 with data from the Swedish Patient Registry, the Swedish Cause of Death Registry and the Swedish Drug Registry using a unique social security number.
RESULTS: Of 623 patients with ATTRv amyloidosis, 288 (46.2%) had received the diagnosis before the age of 70, with an average lookback period of 13.3 years (SD 7.0) and follow-up of 9.0 years (SD 6.0). More than a fifth (21.9%) had their diagnosis of ATTR amyloidosis made while being hospitalised. The most common ATTR-related reasons for healthcare utilisation prior to ATTR amyloidosis diagnosis were neuropathy and heart failure, while the most commonly prescribed medication was antidepressants (19.1%). At the end of follow-up, 30.9% of patients had died at an average age of 69.9 years and an average time of 9.2 years after the diagnosis. Primary cause of death was most often amyloidosis (60.9%) followed by myocardial infarction (8.0%).
CONCLUSIONS: This analysis of patients with ATTRv amyloidosis demonstrates not only the poor outcomes, but also the heterogenous healthcare consumption prior to diagnosis. As effective treatments have become available that significantly slow the progression of the disease, there is an imminent need for increased awareness of the condition and for clinical guidelines that help earlier detection.
METHODS: Data were generated by merging data from the Swedish national quality registry for ATTR amyloidosis (SveATTR) from January 2000 to November 2024 with data from the Swedish Patient Registry, the Swedish Cause of Death Registry and the Swedish Drug Registry using a unique social security number.
RESULTS: Of 623 patients with ATTRv amyloidosis, 288 (46.2%) had received the diagnosis before the age of 70, with an average lookback period of 13.3 years (SD 7.0) and follow-up of 9.0 years (SD 6.0). More than a fifth (21.9%) had their diagnosis of ATTR amyloidosis made while being hospitalised. The most common ATTR-related reasons for healthcare utilisation prior to ATTR amyloidosis diagnosis were neuropathy and heart failure, while the most commonly prescribed medication was antidepressants (19.1%). At the end of follow-up, 30.9% of patients had died at an average age of 69.9 years and an average time of 9.2 years after the diagnosis. Primary cause of death was most often amyloidosis (60.9%) followed by myocardial infarction (8.0%).
CONCLUSIONS: This analysis of patients with ATTRv amyloidosis demonstrates not only the poor outcomes, but also the heterogenous healthcare consumption prior to diagnosis. As effective treatments have become available that significantly slow the progression of the disease, there is an imminent need for increased awareness of the condition and for clinical guidelines that help earlier detection.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE519
Topic
Clinical Outcomes, Economic Evaluation, Health Service Delivery & Process of Care
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory), Neurological Disorders, Rare & Orphan Diseases, Sensory System Disorders (Ear, Eye, Dental, Skin), Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)