PDF(Pubmed)
Abstract：
BACKGROUND: Transthyretin amyloid cardiomyopathy results from the accumulation of wild-type (ATTRwt) or variant (ATTRv) transthyretin amyloid fibrils in the myocardium. THAOS (Transthyretin Amyloidosis Outcomes Survey) is a global, longitudinal, observational survey of patients with ATTRv and ATTRwt amyloidosis and asymptomatic patients with transthyretin mutations.
OBJECTIVE: This study explored temporal trends in ATTRwt amyloidosis diagnoses using data from THAOS.
METHODS: Using THAOS data from December 2007 to January 2020, the following comparisons were made according to year: ATTRwt amyloidosis diagnoses in the United States versus rest of the world, ATTRwt versus ATTRv amyloidosis with cardiac-associated mutations diagnoses, and ATTRwt amyloidosis diagnoses by tissue biopsy versus bone scintigraphy.
RESULTS: There were 1,069 patients with ATTRwt amyloidosis and 525 with ATTRv amyloidosis with cardiac mutations enrolled in THAOS. The median time from symptom onset to ATTRwt amyloidosis diagnosis did not change over the past 5 years (>60 months from 2015-2019). ATTRwt amyloidosis diagnoses increased from 2 in 2005 to >100 per year from 2016, with a more pronounced increase in the United States compared with the rest of the world. Diagnoses of ATTRwt amyloidosis by tissue biopsy increased yearly and peaked in 2014 before declining, whereas diagnoses by bone scintigraphy increased markedly since 2011. ATTRv amyloidosis with cardiac mutation diagnoses increased from 3 in 2005 to 37 in 2011, then plateaued. The proportion of patients with ATTRwt amyloidosis diagnosed with New York Heart Association functional class III/IV heart failure decreased from 2012 (46.4%) to 2019 (16.0%).
CONCLUSIONS: In the past decade, ATTRwt amyloidosis diagnoses increased worldwide. Despite the growing utilization of bone scintigraphy, patients are diagnosed several years after symptom onset. (Transthyretin Amyloidosis Outcomes Survey [THAOS]; NCT00628745).
OBJECTIVE: This study explored temporal trends in ATTRwt amyloidosis diagnoses using data from THAOS.
METHODS: Using THAOS data from December 2007 to January 2020, the following comparisons were made according to year: ATTRwt amyloidosis diagnoses in the United States versus rest of the world, ATTRwt versus ATTRv amyloidosis with cardiac-associated mutations diagnoses, and ATTRwt amyloidosis diagnoses by tissue biopsy versus bone scintigraphy.
RESULTS: There were 1,069 patients with ATTRwt amyloidosis and 525 with ATTRv amyloidosis with cardiac mutations enrolled in THAOS. The median time from symptom onset to ATTRwt amyloidosis diagnosis did not change over the past 5 years (>60 months from 2015-2019). ATTRwt amyloidosis diagnoses increased from 2 in 2005 to >100 per year from 2016, with a more pronounced increase in the United States compared with the rest of the world. Diagnoses of ATTRwt amyloidosis by tissue biopsy increased yearly and peaked in 2014 before declining, whereas diagnoses by bone scintigraphy increased markedly since 2011. ATTRv amyloidosis with cardiac mutation diagnoses increased from 3 in 2005 to 37 in 2011, then plateaued. The proportion of patients with ATTRwt amyloidosis diagnosed with New York Heart Association functional class III/IV heart failure decreased from 2012 (46.4%) to 2019 (16.0%).
CONCLUSIONS: In the past decade, ATTRwt amyloidosis diagnoses increased worldwide. Despite the growing utilization of bone scintigraphy, patients are diagnosed several years after symptom onset. (Transthyretin Amyloidosis Outcomes Survey [THAOS]; NCT00628745).
摘要：
背景：运甲状腺素蛋白淀粉样心肌病是由野生型（ATTRwt）或变体（ATTRv）运甲状腺素蛋白淀粉样原纤维在心肌中的积累引起的。THAOS（转甲状腺素蛋白淀粉样变性结果调查）是一项全球性的，纵向,ATTRv和ATTRwt淀粉样变性患者和甲状腺激素水平突变无症状患者的观察性调查。
目的：本研究使用来自THAOS的数据探索ATTRwt淀粉样变性诊断的时间趋势。
方法：使用2007年12月至2020年1月的THAOS数据，根据年份进行了以下比较：美国与世界其他地区的ATTRwt淀粉样变性诊断，ATTRwt与ATTRv淀粉样变性与心脏相关突变诊断，和ATTRwt淀粉样变性通过组织活检和骨闪烁显像诊断。
结果：有1,069例ATTRwt淀粉样变性和525例ATTRv淀粉样变性和心脏突变患者纳入THAOS。从症状发作到ATTRwt淀粉样变性诊断的中位时间在过去5年中没有变化（2015-2019年>60个月）。ATTRwt淀粉样变性诊断从2005年的2例增加到2016年的每年>100例，与世界其他地区相比，美国的增加更为明显。通过组织活检对ATTRwt淀粉样变性的诊断逐年增加，并在2014年达到峰值，然后下降。而骨闪烁显像诊断自2011年以来显著增加。具有心脏突变的ATTRv淀粉样变性诊断从2005年的3例增加到2011年的37例，然后趋于稳定。从2012年（46.4％）到2019年（16.0％），诊断为纽约心脏协会功能III/IV级心力衰竭的ATTRwt淀粉样变性患者比例下降。
结论：在过去的十年中，ATTRwt淀粉样变性诊断在全球范围内增加。尽管骨闪烁显像的应用越来越多,患者在症状发作几年后被诊断。（转甲状腺素蛋白淀粉样变性结果调查[THAOS]；NCT00628745）。
目的：本研究使用来自THAOS的数据探索ATTRwt淀粉样变性诊断的时间趋势。
方法：使用2007年12月至2020年1月的THAOS数据，根据年份进行了以下比较：美国与世界其他地区的ATTRwt淀粉样变性诊断，ATTRwt与ATTRv淀粉样变性与心脏相关突变诊断，和ATTRwt淀粉样变性通过组织活检和骨闪烁显像诊断。
结果：有1,069例ATTRwt淀粉样变性和525例ATTRv淀粉样变性和心脏突变患者纳入THAOS。从症状发作到ATTRwt淀粉样变性诊断的中位时间在过去5年中没有变化（2015-2019年>60个月）。ATTRwt淀粉样变性诊断从2005年的2例增加到2016年的每年>100例，与世界其他地区相比，美国的增加更为明显。通过组织活检对ATTRwt淀粉样变性的诊断逐年增加，并在2014年达到峰值，然后下降。而骨闪烁显像诊断自2011年以来显著增加。具有心脏突变的ATTRv淀粉样变性诊断从2005年的3例增加到2011年的37例，然后趋于稳定。从2012年（46.4％）到2019年（16.0％），诊断为纽约心脏协会功能III/IV级心力衰竭的ATTRwt淀粉样变性患者比例下降。
结论：在过去的十年中，ATTRwt淀粉样变性诊断在全球范围内增加。尽管骨闪烁显像的应用越来越多,患者在症状发作几年后被诊断。（转甲状腺素蛋白淀粉样变性结果调查[THAOS]；NCT00628745）。
