Efficacy of Tafamidis in Patients With Hereditary and Wild-Type Transthyretin Amyloid Cardiomyopathy

Rapezzi, Claudio; Elliott, Perry; Damy, Thibaud; Nativi-Nicolau, José; Berk, John L.; Velázquez, Eric J.; Boman, Kurt; Gundapaneni, Balarama; Patterson, Terrell A.; Schwartz, Jeffrey H.; Sultan, Marla B.; Maurer, Mathew S.

doi:10.1016/j.jchf.2020.09.011

Cited by 63 publications

(60 citation statements)

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“… Some demographic data have been previously published. 13 6MWT, 6‐minute walk test; ATTRv, variant transthyretin amyloidosis; ATTRwt, wild‐type transthyretin amyloidosis; BMI, body mass index; IVST, interventricular septal wall thickness; KCCQ‐OS, Kansas City Cardiomyopathy Questionnaire Overall Summary; LV, left ventricular; mBMI, modified body mass index; NT‐proBNP, N‐terminal pro‐B‐type natriuretic peptide; NYHA, New York Heart Association; SD, standard deviation. a mBMI is calculated by multiplying the BMI [weight (kg)/height (m 2 )] by serum albumin concentration (g/L).…”

Section: Resultsmentioning

confidence: 99%

“…Some data on the effect of treatment with tafamidis (compared with placebo) on these outcomes have been previously published. 12 , 13 …”

Section: Methodsmentioning

confidence: 99%

“…The decline in KCCQ-OS score over time in ATTR-ACT was more severe in patients with ATTRv and NYHA Class III, although the difference was not large and, as with 6MWT distance, the rate of decline was largely consistent across all Figure 2 All-cause mortality by (A) genotype and (B) NYHA class at baseline. (Some data in patients with ATTRwt and patients with ATTRv have been previously published 13 ). groups.…”

Section: 13mentioning

confidence: 99%

“… 12 Patients were enrolled in ATTR‐ACT for up to 30 months, providing a detailed assessment of patients over a meaningful period of time. Following on from prior publications showing the efficacy of tafamidis in patients with ATTR‐CM, 12 in both patients with ATTRwt and patients with ATTRv, 13 this analysis aims to describe the natural history of ATTR‐CM utilizing data from placebo‐treated patients in ATTR‐ACT.…”

Section: Introductionmentioning

confidence: 99%

“…Figure 4Change in 6MWT distance over time by (A) genotype and (B) NYHA class at baseline. (Some data in patients with ATTRwt and patients with ATTRv have been previously published13 ).…”

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confidence: 99%

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Natural history and progression of transthyretin amyloid cardiomyopathy: insights from ATTR‐ACT

et al. 2021

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Aims Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, fatal disorder that remains underdiagnosed. The Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) was the first large clinical trial to include both wild-type (ATTRwt) and hereditary (ATTRv) patients. A description of the natural history of ATTR-CM, utilizing data from placebo-treated patients in ATTR-ACT, will provide a greater understanding of presentation and progression of ATTR-CM and may aid in disease awareness, earlier diagnosis and treatment monitoring. Methods and results Changes in clinical endpoints (mortality, cardiovascular [CV]-related hospitalizations, 6-min walk test [6MWT] distance and Kansas City Cardiomyopathy Questionnaire Overall Summary [KCCQ-OS] score) from baseline to Month 30 in the 177 patients (134 ATTRwt, 43 ATTRv) who received placebo in ATTR-ACT were assessed. ATTRwt patients tended to have less severe disease at baseline. Over the duration of ATTR-ACT, there were 76 (42.9%) all-cause deaths, and 107 (60.5%) patients had a CV-related hospitalization. There was a lower proportion of all-cause deaths in ATTRwt (49, 36.6%) than ATTRv (27, 62.8%). There was a similar, steady decline in mean (SD) 6MWT distance from baseline to Month 30 in ATTRwt (93.9 [93.7] m) and ATTRv (89.1 [107.2] m) patients. The decline in mean (SD) KCCQ-OS score was less severe in ATTRwt (13.8 [20.7]) than ATTRv (21.0 [26.4]) patients. Conclusions Patients with ATTR-CM experience a severe, progressive disease. In ATTR-ACT, placebo-treated patients with ATTRv, compared with ATTRwt, had more severe disease at baseline, and their disease progressed more rapidly as shown by mortality, hospitalizations and quality of life over time.

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Section: Resultsmentioning

confidence: 99%

“…Some data on the effect of treatment with tafamidis (compared with placebo) on these outcomes have been previously published. 12 , 13 …”

Section: Methodsmentioning

confidence: 99%

Section: 13mentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

“…Figure 4Change in 6MWT distance over time by (A) genotype and (B) NYHA class at baseline. (Some data in patients with ATTRwt and patients with ATTRv have been previously published13 ).…”

mentioning

confidence: 99%

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Natural history and progression of transthyretin amyloid cardiomyopathy: insights from ATTR‐ACT

et al. 2021

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Clinical characteristics, outcome, and therapeutic effect of tafamidis in wild‐type transthyretin amyloid cardiomyopathy

et al. 2023

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Aims Tafamidis improves prognosis in patients with transthyretin amyloid cardiomyopathy (ATTR‐CM). However, real‐world data on the therapeutic effect of tafamidis are lacking. This study aimed to evaluate the clinical course, outcomes, and effectivity monitoring of the therapeutic effect of tafamidis in patients with ATTR‐CM. Methods and results This is a single‐centre, retrospective observational study. We evaluated the clinical characteristics and outcomes in 125 consecutive patients with wild‐type ATTR‐CM (ATTRwt‐CM) treated with tafamidis (treatment group) and 55 untreated patients (treatment‐naïve group). We monitored the therapeutic effect of tafamidis for 12 months by evaluating serial cardiac biomarker and imaging findings. The treatment group had significantly more favourable outcome in all‐cause mortality and hospitalization due to heart failure than the treatment‐naïve group in both the entire cohort (P < 0.01) and the propensity score‐matched cohort (P < 0.05). Kaplan–Meier survival curves showed that tafamidis treatment significantly reduced all‐cause mortality (P = 0.03, log‐rank test), with the curves diverging after approximately 18 months of treatment in the propensity score‐matched cohort. On inverse probability of treatment weighting analysis, tafamidis treatment showed a reduced all‐cause mortality [hazard ratio (HR), 0.31; 95% confidence interval (CI), 0.11–0.93; P = 0.04]. High‐sensitivity cardiac troponin T (hs‐cTnT) > 0.05 ng/mL, B‐type natriuretic peptide (BNP) > 250 pg/mL, and estimated glomerular filtration rate (eGFR) < 45 mL/min/1.73 m2 scored 1 point each. Multivariate logistic regression analysis revealed that a high score (2–3 points) was a significantly poor prognostic factor of composite clinical outcomes, including all‐cause death and hospitalization for heart failure (HR, 1.55; 95% CI, 1.22–1.98; P < 0.01) for patients in the treatment group. After 12 months of tafamidis treatment, hs‐cTnT levels decreased significantly [0.054 (0.036–0.082) vs. 0.044 (0.033–0.076); P = 0.002], with no significant changes in BNP levels, echocardiographic parameters, native T1 value, and extracellular volume fraction on cardiac magnetic resonance imaging. Conclusions The prognosis of patients with ATTRwt‐CM treated with tafamidis was more favourable than that of untreated patients. Patient stratification combined with biomarkers (hs‐cTnT, BNP, and eGFR) predicted clinical outcomes. hs‐cTnT may be a useful biomarker for evaluating the therapeutic effect of tafamidis.

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New effective treatment options reinforce disease awareness: the case of transthyretin cardiac amyloidosis

Perlini

Mussinelli

Salinaro

2021

European J of Heart Fail

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Efficacy of Tafamidis in Patients With Hereditary and Wild-Type Transthyretin Amyloid Cardiomyopathy

Cited by 63 publications

References 29 publications

Natural history and progression of transthyretin amyloid cardiomyopathy: insights from ATTR‐ACT

Natural history and progression of transthyretin amyloid cardiomyopathy: insights from ATTR‐ACT

Clinical characteristics, outcome, and therapeutic effect of tafamidis in wild‐type transthyretin amyloid cardiomyopathy

New effective treatment options reinforce disease awareness: the case of transthyretin cardiac amyloidosis

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