Abstract：
UNASSIGNED: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive disease that causes heart failure due to amyloid fibril deposition. Tafamidis was approved as the first causal treatment in 2020. We here report on real-world data in patients treated with tafamidis for at least 12 months according to the recently defined European Society for Cardiology (ESC) consensus criteria for disease progression.
UNASSIGNED: Three hundred and eight wildtype and 31 hereditary ATTR-CM patients were prospectively enrolled after first diagnosis of ATTR-CM and initiation of tafamidis 61 mg once daily treatment. After 12 months, significant deterioration in Karnofsky Index, estimated glomerular filtration rate (eGFR), N-terminal brain natriuretic peptide (NT-proBNP), septum thickness and left ventricular ejection fraction (LVEF) could be observed, significant disease progression was only detected in 25 patients (9%) using ESC consensus criteria. Mean survival time was 37 months with no differences between responders and non-responders. NT-proBNP was the only independent predictor for poor therapy response (p = .008).
UNASSIGNED: The majority of patients showed no significant disease progression according to the ESC consensus criteria after 12 months of therapy with tafamidis. However, at 12 months, treatment response based on the ESC consensus criteria was not associated with improved survival. Moreover, higher levels of NT-proBNP at diagnosis of ATTR-CM appears to predict poorer treatment response, confirming that timely initiation of therapy is advantageous.
UNASSIGNED: Three hundred and eight wildtype and 31 hereditary ATTR-CM patients were prospectively enrolled after first diagnosis of ATTR-CM and initiation of tafamidis 61 mg once daily treatment. After 12 months, significant deterioration in Karnofsky Index, estimated glomerular filtration rate (eGFR), N-terminal brain natriuretic peptide (NT-proBNP), septum thickness and left ventricular ejection fraction (LVEF) could be observed, significant disease progression was only detected in 25 patients (9%) using ESC consensus criteria. Mean survival time was 37 months with no differences between responders and non-responders. NT-proBNP was the only independent predictor for poor therapy response (p = .008).
UNASSIGNED: The majority of patients showed no significant disease progression according to the ESC consensus criteria after 12 months of therapy with tafamidis. However, at 12 months, treatment response based on the ESC consensus criteria was not associated with improved survival. Moreover, higher levels of NT-proBNP at diagnosis of ATTR-CM appears to predict poorer treatment response, confirming that timely initiation of therapy is advantageous.
摘要：
■转甲状腺素蛋白淀粉样心肌病（ATTR-CM）是一种进行性疾病，由于淀粉样蛋白原纤维沉积而导致心力衰竭。Tafamidis在2020年被批准为第一个因果治疗。根据最近定义的欧洲心脏病学会（ESC）疾病进展共识标准，我们在此报告了使用tafamidis治疗至少12个月的患者的实际数据。
■在首次诊断为ATTR-CM并开始每天一次的塔法米61mg治疗后，前瞻性招募了三百零八名野生型和31名遗传性ATTR-CM患者。12个月后,Karnofsky指数显著恶化，估计肾小球滤过率(eGFR)，N末端脑钠肽（NT-proBNP），可以观察到隔膜厚度和左心室射血分数（LVEF），根据ESC共识标准,仅在25例患者(9%)中检测到显著的疾病进展.平均生存时间为37个月，响应者和非响应者之间没有差异。NT-proBNP是治疗反应不佳的唯一独立预测因子（p=.008）。
■根据ESC共识标准，大多数患者在使用tafamidis治疗12个月后未出现明显的疾病进展。然而,12个月时,基于ESC共识标准的治疗应答与生存率改善无关.此外，在ATTR-CM的诊断中NT-proBNP水平较高似乎预示着较差的治疗反应，确认及时开始治疗是有利的。
■在首次诊断为ATTR-CM并开始每天一次的塔法米61mg治疗后，前瞻性招募了三百零八名野生型和31名遗传性ATTR-CM患者。12个月后,Karnofsky指数显著恶化，估计肾小球滤过率(eGFR)，N末端脑钠肽（NT-proBNP），可以观察到隔膜厚度和左心室射血分数（LVEF），根据ESC共识标准,仅在25例患者(9%)中检测到显著的疾病进展.平均生存时间为37个月，响应者和非响应者之间没有差异。NT-proBNP是治疗反应不佳的唯一独立预测因子（p=.008）。
■根据ESC共识标准，大多数患者在使用tafamidis治疗12个月后未出现明显的疾病进展。然而,12个月时,基于ESC共识标准的治疗应答与生存率改善无关.此外，在ATTR-CM的诊断中NT-proBNP水平较高似乎预示着较差的治疗反应，确认及时开始治疗是有利的。
