René Rettl, Franz Duca, Christina Binder, Theresa-Marie Dachs, Bernhard Cherouny, Luciana Camuz Ligios, Christopher Mann, Lore Schrutka, Daniel Dalos, Silvia Charwat-Resl, Roza Badr Eslam, Johannes Kastner, Diana Bonderman
{"title":"他法非地对转甲状腺素淀粉样心肌病心肌应变的影响。","authors":"René Rettl, Franz Duca, Christina Binder, Theresa-Marie Dachs, Bernhard Cherouny, Luciana Camuz Ligios, Christopher Mann, Lore Schrutka, Daniel Dalos, Silvia Charwat-Resl, Roza Badr Eslam, Johannes Kastner, Diana Bonderman","doi":"10.1080/13506129.2022.2131385","DOIUrl":null,"url":null,"abstract":"<p><strong>Aims: </strong>The impact of tafamidis on myocardial strain in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) have been barely investigated. We aimed to determine tafamidis-induced changes using serial speckle tracking echocardiography and to identify imaging parameters for specific therapy monitoring.</p><p><strong>Methods and results: </strong>ATTR-CM patients underwent serial TTE with two-dimensional (2 D) speckle tracking imaging. Patients receiving tafamidis free acid 61 mg (<i>n</i> = 62) or tafamidis meglumine 20 mg (<i>n</i> = 21) once daily (QD) showed stable measurements at follow-up (61 mg: 8.5 months, 20 mg: 7.0 months) in LV global longitudinal strain (GLS) (61 mg: -11.75% vs. -11.58%, <i>p</i> = 0.534; 20 mg: -10.61% vs. -10.12%, <i>p</i> = 0.309), right ventricular (RV) GLS (61 mg: -14.18% vs. -13.72%, <i>p</i> = 0.377; 20 mg: -14.53% vs. -13.99%, <i>p</i> = 0.452) and left atrial (LA) reservoir strain (LASr; 61 mg: 8.80% vs. 9.42%, <i>p</i> = 0.283; 20 mg: 8.23% vs. 8.67%, <i>p</i> = 0.589), whereas treatment-naïve ATTR-CM patients (<i>n</i> = 54) had clear signs of disease progression at the end of the observation period (10.5 months; LV-GLS: -11.71% vs. -10.59%, <i>p</i> = 0.001; RV-GLS: -14.36% vs. -12.99%, <i>p</i> = 0.038; LASr: 10.67% vs. 8.41%, <i>p</i> = 0.005). Between-group comparison at follow-up revealed beneficial effects of tafamidis free acid 61 mg on LASr (<i>p</i> = 0.003) and the LV (LV-GLS: <i>p</i> = 0.030, interventricular septum (IVS): <i>p</i> = 0.006), resulting in clinical benefits (six-minute walk distance (6-MWD): <i>p</i> = 0.006, NT-proBNP: p= <0.001), while patients treated with tafamidis meglumine 20 mg QD showed positive effects on LASr (<i>p</i> = 0.039), but no differences with respect to the LV (LV-GLS: <i>p</i> = 0.274, IVS: <i>p</i> = 0.068) and clinical status (6-MWD: <i>p</i> = 0.124, NT-proBNP: <i>p</i> = 0.053) compared to the natural course.</p><p><strong>Conclusions: </strong>Treatment with tafamidis free acid 61 mg in ATTR-CM patients delays the deterioration of LA and LV longitudinal function, resulting in significant clinical benefits compared with natural history. Serial TTE with 2 D speckle tracking imaging may be appropriate for disease-specific therapy monitoring.</p>","PeriodicalId":50964,"journal":{"name":"Amyloid-Journal of Protein Folding Disorders","volume":null,"pages":null},"PeriodicalIF":5.2000,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"8","resultStr":"{\"title\":\"Impact of tafamidis on myocardial strain in transthyretin amyloid cardiomyopathy.\",\"authors\":\"René Rettl, Franz Duca, Christina Binder, Theresa-Marie Dachs, Bernhard Cherouny, Luciana Camuz Ligios, Christopher Mann, Lore Schrutka, Daniel Dalos, Silvia Charwat-Resl, Roza Badr Eslam, Johannes Kastner, Diana Bonderman\",\"doi\":\"10.1080/13506129.2022.2131385\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Aims: </strong>The impact of tafamidis on myocardial strain in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) have been barely investigated. We aimed to determine tafamidis-induced changes using serial speckle tracking echocardiography and to identify imaging parameters for specific therapy monitoring.</p><p><strong>Methods and results: </strong>ATTR-CM patients underwent serial TTE with two-dimensional (2 D) speckle tracking imaging. Patients receiving tafamidis free acid 61 mg (<i>n</i> = 62) or tafamidis meglumine 20 mg (<i>n</i> = 21) once daily (QD) showed stable measurements at follow-up (61 mg: 8.5 months, 20 mg: 7.0 months) in LV global longitudinal strain (GLS) (61 mg: -11.75% vs. -11.58%, <i>p</i> = 0.534; 20 mg: -10.61% vs. -10.12%, <i>p</i> = 0.309), right ventricular (RV) GLS (61 mg: -14.18% vs. -13.72%, <i>p</i> = 0.377; 20 mg: -14.53% vs. -13.99%, <i>p</i> = 0.452) and left atrial (LA) reservoir strain (LASr; 61 mg: 8.80% vs. 9.42%, <i>p</i> = 0.283; 20 mg: 8.23% vs. 8.67%, <i>p</i> = 0.589), whereas treatment-naïve ATTR-CM patients (<i>n</i> = 54) had clear signs of disease progression at the end of the observation period (10.5 months; LV-GLS: -11.71% vs. -10.59%, <i>p</i> = 0.001; RV-GLS: -14.36% vs. -12.99%, <i>p</i> = 0.038; LASr: 10.67% vs. 8.41%, <i>p</i> = 0.005). Between-group comparison at follow-up revealed beneficial effects of tafamidis free acid 61 mg on LASr (<i>p</i> = 0.003) and the LV (LV-GLS: <i>p</i> = 0.030, interventricular septum (IVS): <i>p</i> = 0.006), resulting in clinical benefits (six-minute walk distance (6-MWD): <i>p</i> = 0.006, NT-proBNP: p= <0.001), while patients treated with tafamidis meglumine 20 mg QD showed positive effects on LASr (<i>p</i> = 0.039), but no differences with respect to the LV (LV-GLS: <i>p</i> = 0.274, IVS: <i>p</i> = 0.068) and clinical status (6-MWD: <i>p</i> = 0.124, NT-proBNP: <i>p</i> = 0.053) compared to the natural course.</p><p><strong>Conclusions: </strong>Treatment with tafamidis free acid 61 mg in ATTR-CM patients delays the deterioration of LA and LV longitudinal function, resulting in significant clinical benefits compared with natural history. Serial TTE with 2 D speckle tracking imaging may be appropriate for disease-specific therapy monitoring.</p>\",\"PeriodicalId\":50964,\"journal\":{\"name\":\"Amyloid-Journal of Protein Folding Disorders\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":5.2000,\"publicationDate\":\"2023-03-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"8\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Amyloid-Journal of Protein Folding Disorders\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1080/13506129.2022.2131385\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"BIOCHEMISTRY & MOLECULAR BIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Amyloid-Journal of Protein Folding Disorders","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/13506129.2022.2131385","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"BIOCHEMISTRY & MOLECULAR BIOLOGY","Score":null,"Total":0}
引用次数: 8
摘要
目的:他法非地对转甲状腺素淀粉样心肌病(atr - cm)患者心肌应变的影响研究甚少。我们的目的是利用序列斑点跟踪超声心动图确定他法底炎引起的变化，并确定特定治疗监测的成像参数。方法和结果:atr - cm患者采用二维(2d)斑点跟踪成像进行连续TTE治疗。每日一次(QD)接受他非他胺酸61mg (n = 62)或他非他胺meglumine 20mg (n = 21)的患者在随访时显示LV整体纵向张力(GLS)的稳定测量(61mg: 8.5个月，20mg: 7.0个月)(61mg: -11.75% vs -11.58%， p = 0.534;20毫克:-10.61%比-10.12%,p = 0.309),右心室(RV) gl (61 mg: -14.18%比-13.72%,p = 0.377;20 mg: -14.53% vs. -13.99%， p = 0.452)和左心房(LA)储层应变(LASr;61 mg: 8.80% vs. 9.42%， p = 0.283;20 mg: 8.23% vs. 8.67%， p = 0.589)，而treatment-naïve atr - cm患者(n = 54)在观察结束时(10.5个月;LV-GLS: -11.71% vs. -10.59%， p = 0.001;RV-GLS: -14.36% vs. -12.99%， p = 0.038;LASr: 10.67% vs. 8.41%， p = 0.005)。群体间的比较在随访显示有益的tafamidis游离酸61毫克LASr (p = 0.003)和LV (LV-GLS: p = 0.030,室间隔(IVS): p = 0.006),导致临床益处(6分钟步行距离(6-MWD): p = 0.006,中位数水平以上病人:p = p = 0.039),但没有差异对LV (LV-GLS: p = 0.274,静脉注射:p = 0.068)和临床状态(6-MWD: p = 0.124,中位数水平以上病人:p = 0.053)相比,自然。结论:atr - cm患者使用他法非地酸61 mg治疗可延缓左室和左室纵向功能的恶化，与自然史相比，临床获益显著。串行TTE与二维散斑跟踪成像可能适用于疾病特异性治疗监测。
Impact of tafamidis on myocardial strain in transthyretin amyloid cardiomyopathy.
Aims: The impact of tafamidis on myocardial strain in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) have been barely investigated. We aimed to determine tafamidis-induced changes using serial speckle tracking echocardiography and to identify imaging parameters for specific therapy monitoring.
Methods and results: ATTR-CM patients underwent serial TTE with two-dimensional (2 D) speckle tracking imaging. Patients receiving tafamidis free acid 61 mg (n = 62) or tafamidis meglumine 20 mg (n = 21) once daily (QD) showed stable measurements at follow-up (61 mg: 8.5 months, 20 mg: 7.0 months) in LV global longitudinal strain (GLS) (61 mg: -11.75% vs. -11.58%, p = 0.534; 20 mg: -10.61% vs. -10.12%, p = 0.309), right ventricular (RV) GLS (61 mg: -14.18% vs. -13.72%, p = 0.377; 20 mg: -14.53% vs. -13.99%, p = 0.452) and left atrial (LA) reservoir strain (LASr; 61 mg: 8.80% vs. 9.42%, p = 0.283; 20 mg: 8.23% vs. 8.67%, p = 0.589), whereas treatment-naïve ATTR-CM patients (n = 54) had clear signs of disease progression at the end of the observation period (10.5 months; LV-GLS: -11.71% vs. -10.59%, p = 0.001; RV-GLS: -14.36% vs. -12.99%, p = 0.038; LASr: 10.67% vs. 8.41%, p = 0.005). Between-group comparison at follow-up revealed beneficial effects of tafamidis free acid 61 mg on LASr (p = 0.003) and the LV (LV-GLS: p = 0.030, interventricular septum (IVS): p = 0.006), resulting in clinical benefits (six-minute walk distance (6-MWD): p = 0.006, NT-proBNP: p= <0.001), while patients treated with tafamidis meglumine 20 mg QD showed positive effects on LASr (p = 0.039), but no differences with respect to the LV (LV-GLS: p = 0.274, IVS: p = 0.068) and clinical status (6-MWD: p = 0.124, NT-proBNP: p = 0.053) compared to the natural course.
Conclusions: Treatment with tafamidis free acid 61 mg in ATTR-CM patients delays the deterioration of LA and LV longitudinal function, resulting in significant clinical benefits compared with natural history. Serial TTE with 2 D speckle tracking imaging may be appropriate for disease-specific therapy monitoring.
期刊介绍：
Amyloid: the Journal of Protein Folding Disorders is dedicated to the study of all aspects of the protein groups and associated disorders that are classified as the amyloidoses as well as other disorders associated with abnormal protein folding. The journals major focus points are:
etiology,
pathogenesis,
histopathology,
chemical structure,
nature of fibrillogenesis;
whilst also publishing papers on the basic and chemical genetic aspects of many of these disorders.
Amyloid is recognised as one of the leading publications on amyloid protein classifications and the associated disorders, as well as clinical studies on all aspects of amyloid related neurodegenerative diseases and major clinical studies on inherited amyloidosis, especially those related to transthyretin. The Journal also publishes book reviews, meeting reports, editorials, thesis abstracts, review articles and symposia in the various areas listed above.
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