Karan Shahi MSc , Robert J.H. Miller MD , Steven Dykstra PhD , Yuanchao Feng PhD , Jonathan G. Howlett MD , Victor Jimenez-Zepeda MD , Jan Veenhuyzen RN, BScN , James A. White MD , Nowell M. Fine MD, SM
{"title":"多心脏生物标志物在经甲状腺蛋白淀粉样变性心肌病患者治疗与未治疗他法非地的纵向变化","authors":"Karan Shahi MSc , Robert J.H. Miller MD , Steven Dykstra PhD , Yuanchao Feng PhD , Jonathan G. Howlett MD , Victor Jimenez-Zepeda MD , Jan Veenhuyzen RN, BScN , James A. White MD , Nowell M. Fine MD, SM","doi":"10.1016/j.cjco.2025.05.012","DOIUrl":null,"url":null,"abstract":"<div><h3>Background</h3><div>Tafamidis is an oral transthyretin stabilizer that improves survival in transthyretin amyloidosis cardiomyopathy (ATTR-CM), but only limited real-world data describe serial cardiac biomarker changes following treatment initiation. The primary objective of this study was to characterize longitudinal changes across multiple cardiac biomarker domains in tafamidis-treated ATTR-CM patients, to describe how these parameters evolve over time in routine clinical practice. We also report the same outcomes in untreated patients to reflect the natural disease history in a modern real-world cohort.</div></div><div><h3>Methods</h3><div>Clinical, biochemical, and cardiac imaging parameters were serially assessed at baseline and 1-year follow-up for 145 ATTR-CM patients, both those treated and those untreated with tafamidis.</div></div><div><h3>Results</h3><div>The median age was 80 years (range: 73-86), and 80 patients (55%) received tafamidis. At baseline, the treated group was younger and exhibited less advanced disease, relative to the untreated group. Treatment with tafamadis was associated with stabilization in N-terminal pro-B-type natriuretic peptide (NTproBNP) level, troponin-T level, and New York Heart Association functional class at 1-year follow-up, whereas the untreated group demonstrated worsening (all comparisons <em>P</em> < 0.05). Tafamidis treatment status was not significantly associated with National Amyloidosis Center or Mayo Clinic disease stage.</div></div><div><h3>Conclusions</h3><div>NTproBNP level, troponin-T level, and New York Heart Association functional class remain stable over 1 year in a real-world cohort of tafamidis-treated ATTR-CM patients. These results may help inform therapeutic monitoring strategies in clinical practice.</div></div>","PeriodicalId":36924,"journal":{"name":"CJC Open","volume":"7 9","pages":"Pages 1190-1197"},"PeriodicalIF":2.5000,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Longitudinal Changes in Multiple Cardiac Biomarkers in Transthyretin Amyloidosis Cardiomyopathy Patients Treated Vs Untreated with Tafamidis\",\"authors\":\"Karan Shahi MSc , Robert J.H. Miller MD , Steven Dykstra PhD , Yuanchao Feng PhD , Jonathan G. Howlett MD , Victor Jimenez-Zepeda MD , Jan Veenhuyzen RN, BScN , James A. White MD , Nowell M. Fine MD, SM\",\"doi\":\"10.1016/j.cjco.2025.05.012\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Background</h3><div>Tafamidis is an oral transthyretin stabilizer that improves survival in transthyretin amyloidosis cardiomyopathy (ATTR-CM), but only limited real-world data describe serial cardiac biomarker changes following treatment initiation. The primary objective of this study was to characterize longitudinal changes across multiple cardiac biomarker domains in tafamidis-treated ATTR-CM patients, to describe how these parameters evolve over time in routine clinical practice. We also report the same outcomes in untreated patients to reflect the natural disease history in a modern real-world cohort.</div></div><div><h3>Methods</h3><div>Clinical, biochemical, and cardiac imaging parameters were serially assessed at baseline and 1-year follow-up for 145 ATTR-CM patients, both those treated and those untreated with tafamidis.</div></div><div><h3>Results</h3><div>The median age was 80 years (range: 73-86), and 80 patients (55%) received tafamidis. At baseline, the treated group was younger and exhibited less advanced disease, relative to the untreated group. Treatment with tafamadis was associated with stabilization in N-terminal pro-B-type natriuretic peptide (NTproBNP) level, troponin-T level, and New York Heart Association functional class at 1-year follow-up, whereas the untreated group demonstrated worsening (all comparisons <em>P</em> < 0.05). Tafamidis treatment status was not significantly associated with National Amyloidosis Center or Mayo Clinic disease stage.</div></div><div><h3>Conclusions</h3><div>NTproBNP level, troponin-T level, and New York Heart Association functional class remain stable over 1 year in a real-world cohort of tafamidis-treated ATTR-CM patients. These results may help inform therapeutic monitoring strategies in clinical practice.</div></div>\",\"PeriodicalId\":36924,\"journal\":{\"name\":\"CJC Open\",\"volume\":\"7 9\",\"pages\":\"Pages 1190-1197\"},\"PeriodicalIF\":2.5000,\"publicationDate\":\"2025-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"CJC Open\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S2589790X25003932\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"CARDIAC & CARDIOVASCULAR SYSTEMS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"CJC Open","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2589790X25003932","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"CARDIAC & CARDIOVASCULAR SYSTEMS","Score":null,"Total":0}
Longitudinal Changes in Multiple Cardiac Biomarkers in Transthyretin Amyloidosis Cardiomyopathy Patients Treated Vs Untreated with Tafamidis
Background
Tafamidis is an oral transthyretin stabilizer that improves survival in transthyretin amyloidosis cardiomyopathy (ATTR-CM), but only limited real-world data describe serial cardiac biomarker changes following treatment initiation. The primary objective of this study was to characterize longitudinal changes across multiple cardiac biomarker domains in tafamidis-treated ATTR-CM patients, to describe how these parameters evolve over time in routine clinical practice. We also report the same outcomes in untreated patients to reflect the natural disease history in a modern real-world cohort.
Methods
Clinical, biochemical, and cardiac imaging parameters were serially assessed at baseline and 1-year follow-up for 145 ATTR-CM patients, both those treated and those untreated with tafamidis.
Results
The median age was 80 years (range: 73-86), and 80 patients (55%) received tafamidis. At baseline, the treated group was younger and exhibited less advanced disease, relative to the untreated group. Treatment with tafamadis was associated with stabilization in N-terminal pro-B-type natriuretic peptide (NTproBNP) level, troponin-T level, and New York Heart Association functional class at 1-year follow-up, whereas the untreated group demonstrated worsening (all comparisons P < 0.05). Tafamidis treatment status was not significantly associated with National Amyloidosis Center or Mayo Clinic disease stage.
Conclusions
NTproBNP level, troponin-T level, and New York Heart Association functional class remain stable over 1 year in a real-world cohort of tafamidis-treated ATTR-CM patients. These results may help inform therapeutic monitoring strategies in clinical practice.