Clinical manifestation, economic burden, and mortality in patients with transthyretin cardiac amyloidosis

• Published in: Orphanet journal of rare diseases Vol. 17; no. 1; pp. 1 - 262
• Main Authors: Jang, Suk-Chan, Nam, Jin Hyun, Lee, Seung-Ah, An, Dasom, Kim, Hye-Lin, Kwon, Sun-Hong, Lee, Eui-Kyung
• Format: Journal Article
• Language: English
• Published: London BioMed Central Ltd 15-07-2022; BioMed Central; BMC
• Subjects: Age; Amyloidosis; Analysis; ATTR-CM; Cardiac amyloidosis; Cardiomyopathy; Codes; Comorbidity; Coronary artery disease; Costs; Diagnosis; Diagnostic tests; Disease; Economic burden; Health care; Health services utilization; Heart diseases; Heart failure; Hospitals; Life expectancy; Liver transplants; Medical care; Medical care, Cost of; Medical diagnosis; Medical prognosis; Medical research; Mortality; Nervous system; Patient outcomes; Patients; Population; Population studies; Rare diseases; Resource utilization; Risk factors; Survival; Survival analysis; Survival rates; Transthyretin; Utilization; South Korea; United States > US
• ISSN: 1750-1172; 1750-1172
• DOI: 10.1186/s13023-022-02425-3

Transthyretin cardiac amyloidosis, also known as transthyretin cardiomyopathy (ATTR-CM) is a poorly-recognized disease with delayed diagnosis and poor prognosis. This nationwide population-based study aimed to identify disease manifestations, economic burden, and mortality of patients with ATTR-CM. Data of newly diagnosed patients with ATTR-CM between 2013 and 2018 from the Korean National Health Insurance Service were used, covering the entire population. Patient characteristics included comorbidities, medical procedures, and medication. Healthcare resource utilization and medical costs were observed as measures of the economic burden. The Kaplan-Meier survival curve and years of potential life lost (YPLL) from the general population were estimated for disease burden with ATTR CM. A total of 175 newly diagnosed patients with ATTR-CM were identified. The most common cardiac manifestation was hypertension (51.3%), while the most common non-cardiac manifestation was musculoskeletal disease (68.0%). Mean medical costs at the post-cohort entry date were significantly higher than those at the pre-cohort entry date ($1,864 vs. $400 per patient per month (PPPM), p < 0.001). Of the total medical costs during the study period, the proportion of inpatients cost was 12.9 times higher than the outpatients cost ($1,730 and $134 PPPM, respectively). The median survival time was 3.53 years from the first diagnosis of ATTR-CM, and the mean (SD) YPLL was 13.0 (7.7).