School of Pharmacy, Sungkyunkwan University, 2066 Seobu-ro, Jangan-gu, Suwon, Gyeonggi-do, Republic of Korea.
Division of Big Data Science, Korea University Sejong Campus, Sejong, Republic of Korea.
Orphanet J Rare Dis. 2022 Jul 15;17(1):262. 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).
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.
转甲状腺素蛋白心脏淀粉样变，又称转甲状腺素蛋白心肌病（ATTR-CM），是一种诊断延迟、预后不良的疾病。本项全国性基于人群的研究旨在确定ATTR-CM 患者的疾病表现、经济负担和死亡率。
使用韩国国家健康保险服务 2013 年至 2018 年间新诊断为 ATTR-CM 的患者数据，覆盖全部人群。患者特征包括合并症、医疗程序和药物治疗。观察医疗资源利用和医疗费用作为经济负担的衡量标准。使用 Kaplan-Meier 生存曲线和普通人群的潜在寿命损失年（YPLL）来估计ATTR-CM 的疾病负担。
共确定了 175 例新诊断的 ATTR-CM 患者。最常见的心脏表现是高血压（51.3%），而最常见的非心脏表现是肌肉骨骼疾病（68.0%）。队列入组后日期的平均医疗费用明显高于队列入组前日期（每月每位患者分别为 1864 韩元与 400 韩元，p<0.001）。在研究期间的总医疗费用中，住院患者的费用比例比门诊患者高 12.9 倍（分别为 1730 韩元与 134 韩元/月/人）。从 ATTR-CM 的首次诊断起，中位生存时间为 3.53 年，平均（SD）YPLL 为 13.0（7.7）年。
ATTR-CM 患者的生存时间短，医疗费用高。为了降低临床和经济负担，仔细检查患者的疾病表现有助于早期诊断和治疗。
