Design and Rationale of the Phase 3 ATTR-ACT Clinical Trial (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial)

Maurer, Mathew S.; Elliott, Perry; Merlini, Giampaolo; Shah, Sanjiv J.; Cruz, Márcia Waddington; Flynn, Alison; Gundapaneni, Balarama; Hahn, C.; Riley, Steven; Schwartz, Jeffrey H.; Sultan, Marla B.; Rapezzi, Claudio

doi:10.1161/circheartfailure.116.003815

Cited by 66 publications

(60 citation statements)

References 54 publications

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“…[21][22][23] The median age of TTR-CA patients in the Transthyretin Amyloidosis Cardiomyopathy Clinical Trial was 74 years. 24 Thus, we enrolled patients aged ≥70 years in this study.…”

Section: Predictive Impact Of Our Index For 99 M Tc-pyp Positivitymentioning

confidence: 99%

Combination of Commonly Examined Parameters Is a Useful Predictor of Positive <sup>99 m</sup>Tc-Labeled Pyrophosphate Scintigraphy Findings in Elderly Patients With Suspected Transthyretin Cardiac Amyloidosis

Marume

Takashio

Nishi

et al. 2019

Circ J

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potential benefit from emerging therapies. 2 It has been reported that 13% of elderly patients (mean age, 82 years) who have heart failure with preserved ejection fraction (HFpEF) are diagnosed with ATTRwt. 3 Several postmortem studies found cardiac amyloid deposition in up to 25% of individuals over 80 years of age. 4 The precise diagnosis of CA requires endomyocardial biopsy (EMB) to demonstrate disease-specific deposition. However, EMB is a relatively invasive procedure 5 and so cannot be performed routinely, especially in elderly patients. A recent systematic evaluation of bone scintigraphy C ardiac amyloidosis (CA) is a secondary cardiomyopathy with a hypertrophic appearance caused by deposition of anomalous fibrillar proteins in the myocardial extracellular matrix that originated from a precursor-altered protein called amyloid. 1 There are 3 main types of CA: acquired monoclonal immunoglobulin light chain amyloidosis (AL amyloidosis); mutant transthyretin (TTR) amyloidosis (ATTRm); and wild-type transthyretin amyloidosis (ATTRwt). ATTRwt is becoming increasingly recognized because of aging of populations, advancements in the understanding of the disease's pathobiology, and the

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“…[21][22][23] The median age of TTR-CA patients in the Transthyretin Amyloidosis Cardiomyopathy Clinical Trial was 74 years. 24 Thus, we enrolled patients aged ≥70 years in this study.…”

Section: Predictive Impact Of Our Index For 99 M Tc-pyp Positivitymentioning

confidence: 99%

Combination of Commonly Examined Parameters Is a Useful Predictor of Positive <sup>99 m</sup>Tc-Labeled Pyrophosphate Scintigraphy Findings in Elderly Patients With Suspected Transthyretin Cardiac Amyloidosis

Marume

Takashio

Nishi

et al. 2019

Circ J

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“…We excluded patients with other form of amyloid cardiomyopathy, as well as those ATTR‐AC subjects enrolled in the ATTR‐ACT trial (29 patients enrolled in Bologna, Florence and Pavia). ATTRv‐AC patients with a prevalent neurological phenotype were also excluded according to the ATTR‐ACT protocol . Participating centres were requested to include main patients' characteristics at first clinical evaluation in a centralized anonymized database.…”

Section: Characteristics Of Transthyretin Amyloid Cardiomyopathy Triamentioning

confidence: 99%

Real‐world versus trial patients with transthyretin amyloid cardiomyopathy

Canepa

Tini

Musumeci

et al. 2019

European J of Heart Fail

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“…Diflunisal, a nonsteroidal anti-inflammatory, has been shown to improve neurological symptoms and Tafamidis has been demonstrated to delay disease progression 43 in familiar peripheral neuropathy caused by TTRv . Ongoing trials are evaluating the effect on the heart of Tafamidis 44 . Only liver transplant for ATTRv-CA and treatment of hematologic monoclonal disorder, in AL-CA, is currently approved.…”

Section: Extracellular Deposit Storagementioning

confidence: 99%

Storage diseases with hypertrophic cardiomyopathy phenotype

Ruiz-Guerrero

Barriales‐Villa

2018

gcsp

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Never judge a book by its cover, nor assume hypertrophic cardiomyopathy (HCM) as sarcomeric, as appearances can deceive. HCM phenocopies account for a 5–10% of the cases, mainly represented by storage diseases, flagged by the increasing prevalence of senile cardiac amyloid in developing countries. Multisystemic and heterogeneous presentation of these entities is a challenge for clinicians, and time delay in diagnosis is a major concern. Promising drugs and gene-specific tailored therapies are under development, therefore, more than ever, appropriate understanding of these conditions is mandatory for adequate early treatment and counselling.In this review, storage disorders will be classified as extracellular and intracellular deposit storage diseases, focusing our attention on the most prevalent conditions from the cardiologist’s perspective.

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Design and Rationale of the Phase 3 ATTR-ACT Clinical Trial (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial)

Abstract: URL: http://www.clinicaltrials.gov. Unique identifier: NCT01994889.

Cited by 66 publications

References 54 publications

Combination of Commonly Examined Parameters Is a Useful Predictor of Positive <sup>99 m</sup>Tc-Labeled Pyrophosphate Scintigraphy Findings in Elderly Patients With Suspected Transthyretin Cardiac Amyloidosis

Combination of Commonly Examined Parameters Is a Useful Predictor of Positive <sup>99 m</sup>Tc-Labeled Pyrophosphate Scintigraphy Findings in Elderly Patients With Suspected Transthyretin Cardiac Amyloidosis

Real‐world versus trial patients with transthyretin amyloid cardiomyopathy

Storage diseases with hypertrophic cardiomyopathy phenotype

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