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

Abstract Background 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 pat...

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Main Authors: Suk-Chan Jang, Jin Hyun Nam, Seung-Ah Lee, Dasom An, Hye-Lin Kim, Sun-Hong Kwon, Eui-Kyung Lee
Format: Article
Language:English
Published: BMC 2022-07-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Online Access:https://doi.org/10.1186/s13023-022-02425-3
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author Suk-Chan Jang
Jin Hyun Nam
Seung-Ah Lee
Dasom An
Hye-Lin Kim
Sun-Hong Kwon
Eui-Kyung Lee
author_facet Suk-Chan Jang
Jin Hyun Nam
Seung-Ah Lee
Dasom An
Hye-Lin Kim
Sun-Hong Kwon
Eui-Kyung Lee
author_sort Suk-Chan Jang
collection DOAJ
description Abstract Background 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. Methods 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. Results 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). Conclusions Patients with ATTR-CM had short survival and high medical costs. To reduce the clinical and economic burdens, carefully examining manifestations of disease in patients can help with early diagnosis and treatment.
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spelling doaj.art-97606425260840bc8a47904f5a7b30522022-12-22T02:11:49ZengBMCOrphanet Journal of Rare Diseases1750-11722022-07-0117111010.1186/s13023-022-02425-3Clinical manifestation, economic burden, and mortality in patients with transthyretin cardiac amyloidosisSuk-Chan Jang0Jin Hyun Nam1Seung-Ah Lee2Dasom An3Hye-Lin Kim4Sun-Hong Kwon5Eui-Kyung Lee6School of Pharmacy, Sungkyunkwan UniversityDivision of Big Data Science, Korea University Sejong CampusDivision of Cardiology, Department of Internal Medicine, Asan Medical Center, University of Ulsan College of MedicineSchool of Pharmacy, Sungkyunkwan UniversityCollege of Pharmacy, Sahmyook UniversitySchool of Pharmacy, Sungkyunkwan UniversitySchool of Pharmacy, Sungkyunkwan UniversityAbstract Background 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. Methods 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. Results 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). Conclusions Patients with ATTR-CM had short survival and high medical costs. To reduce the clinical and economic burdens, carefully examining manifestations of disease in patients can help with early diagnosis and treatment.https://doi.org/10.1186/s13023-022-02425-3ATTR-CMCardiac amyloidosisEconomic burdenHeart failureSurvival rates
spellingShingle Suk-Chan Jang
Jin Hyun Nam
Seung-Ah Lee
Dasom An
Hye-Lin Kim
Sun-Hong Kwon
Eui-Kyung Lee
Clinical manifestation, economic burden, and mortality in patients with transthyretin cardiac amyloidosis
Orphanet Journal of Rare Diseases
ATTR-CM
Cardiac amyloidosis
Economic burden
Heart failure
Survival rates
title Clinical manifestation, economic burden, and mortality in patients with transthyretin cardiac amyloidosis
title_full Clinical manifestation, economic burden, and mortality in patients with transthyretin cardiac amyloidosis
title_fullStr Clinical manifestation, economic burden, and mortality in patients with transthyretin cardiac amyloidosis
title_full_unstemmed Clinical manifestation, economic burden, and mortality in patients with transthyretin cardiac amyloidosis
title_short Clinical manifestation, economic burden, and mortality in patients with transthyretin cardiac amyloidosis
title_sort clinical manifestation economic burden and mortality in patients with transthyretin cardiac amyloidosis
topic ATTR-CM
Cardiac amyloidosis
Economic burden
Heart failure
Survival rates
url https://doi.org/10.1186/s13023-022-02425-3
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