3.8 Review

Multidisciplinary Approaches for Transthyretin Amyloidosis

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Transthyretin cardiac amyloidosis: A treatable form of heart failure with a preserved ejection fraction

Jan M. Griffin et al.

Summary: Cardiac amyloidosis is a rare disease with limited therapeutic options, but advancements in diagnostic modalities and emerging therapies are challenging the traditional beliefs about its prognosis. Early recognition and prompt initiation of novel agents are crucial in improving the outcomes for patients with ATTR-CA.

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Early changes of nerve integrity in preclinical carriers of hereditary transthyretin Ala117Ser amyloidosis with polyneuropathy

Ming-Chang Chiang et al.

Summary: This study found that in late-onset ATTRv-PN carriers, median conduction parameters were the most common neurophysiological abnormalities and served as surrogate signatures of small- and large-fiber impairment. The combination of median distal motor latency and IENF density can reflect early neuropathy in carriers.

EUROPEAN JOURNAL OF NEUROLOGY (2021)

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Prevalence of transthyretin amyloid cardiomyopathy in male patients who underwent bilateral carpal tunnel surgery: The ACTUAL study

Pier Filippo Vianello et al.

Summary: Bilateral carpal tunnel syndrome patients, particularly those with left ventricular hypertrophy, may have a higher risk of transthyretin cardiac amyloidosis. Screening with electrocardiography and echocardiography appeared feasible and effective, especially in patients with LVH. Early detection of the disease may be achieved through this method.

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Transthyretin amyloid deposits in lumbar spinal stenosis and assessment of signs of systemic amyloidosis

P. Eldhagen et al.

Summary: The study found a high prevalence of ATTR deposits in ligament tissue of patients undergoing surgery for lumbar spinal stenosis, but these deposits were not associated with manifest ATTR cardiac amyloidosis. The presence of fibril type A, a history of carpal tunnel syndrome, and ATTR deposits in surrounding tissues suggest that amyloid deposits in ligament tissue may be an early manifestation of systemic ATTR disease.

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Tenosynovial complications identify TTR cardiac amyloidosis among patients with hypertrophic cardiomyopathy phenotype

F. Cappelli et al.

Summary: Recent evidence suggests that carpal tunnel syndrome (CTS) and brachial biceps tendon rupture (BBTR) are red flags for ATTR cardiac amyloidosis (ATTR-CA), with the prevalence of CTS and BBTR being significantly higher in ATTR-CA patients compared to patients with other hypertrophic phenotypes like HCM or AFD. The study concluded that the presence of bilateral CTS in ATTR-CA patients had the highest positive predictive accuracy for ATTR-CA, making upper limb assessment a simple and effective way to raise suspicion of ATTR-CA in patients with HCM phenotypes.

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Two distinct mechanisms of neuropathy in immunoglobulin light chain (AL) amyloidosis

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Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

David Adams et al.

Summary: Hereditary transthyretin-mediated amyloidosis is a rare genetic disease caused by mutations in the TTR gene. Long-term treatment with patisiran, an RNA interference therapeutic, showed sustained efficacy and acceptable safety profile in patients with this condition, as demonstrated in a 12-month interim analysis of a global open-label extension study.

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Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data

Nicholas J. Viney et al.

Summary: AKCEA-TTR-L-Rx, a ligand-conjugated antisense drug, showed significantly increased potency compared to unconjugated inotersen in human hepatocyte cell culture and transgenic hTTR mice expressing a mutated human genomic TTR sequence. In a phase 1 study with healthy volunteers, AKCEA-TTR-L-Rx demonstrated a reduction in TTR levels and a favorable safety profile, supporting its further development for the treatment of ATTR polyneuropathy and cardiomyopathy.

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Impact of Tafamidis on Health-Related Quality of Life in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial)

Mazen Hanna et al.

Summary: In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial, tafamidis significantly reduced mortality and cardiovascular-related hospitalizations, while improving patient-reported quality of life measures, demonstrating its effectiveness in patients with ATTR-CM.

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Diagnosis and treatment of cardiac amyloidosis: a position statement of the ESC Working Group on Myocardial and Pericardial Diseases

Pablo Garcia-Pavia et al.

Summary: Cardiac amyloidosis is a serious infiltrative disease caused by the deposition of amyloid fibrils in the heart, which can be due to genetic variants or acquired conditions. With advances in imaging techniques, non-invasive diagnosis is now possible, showing that cardiac amyloidosis is more common than previously thought. The Working Group on Myocardial and Pericardial Disease has proposed definitions and diagnostic algorithms for cardiac amyloidosis.

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Covid-19 in liver transplant recipients: the French SOT COVID registry

Jerome Dumortier et al.

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Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Teresa Coelho et al.

Summary: AKCEA-TTR-L-Rx is a ligand-conjugated antisense drug being developed for the treatment of hereditary transthyretin amyloidosis. It utilizes a triantennary N-acetylgalactosamine moiety to increase drug potency for lower and less frequent dosing. The NEURO-TTRansform study aims to evaluate the efficacy of AKCEA-TTR-L-Rx in improving neurologic function and quality of life for hATTR-PN patients.

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Diagnosis and treatment of cardiac amyloidosis: a position statement of the ESC Working Group on Myocardial and Pericardial Diseases

Pablo Garcia-Pavia et al.

Summary: Cardiac amyloidosis is a serious and progressive infiltrative disease caused by amyloid fibril deposition in the heart. Advances in imaging techniques and diagnostic algorithms have made it possible to diagnose the condition non-invasively. Monitoring and treating cardiac amyloidosis are important in bridging the gap between research advances and clinical practice.

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Inotersen therapy of transthyretin amyloid cardiomyopathy

Noel R. Dasgupta et al.

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Diagnostic Accuracy of [11C]PIB Positron Emission Tomography for Detection of Cardiac Amyloidosis

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Amyloid nomenclature 2020: update and recommendations by the International Society of Amyloidosis (ISA) nomenclature committee

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Association of the V122I Hereditary Transthyretin Amyloidosis Genetic Variant With Heart Failure Among Individuals of African or Hispanic/Latino Ancestry

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Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

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Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years

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Cutaneous Nerve Biomarkers in Transthyretin Familial Amyloid Polyneuropathy

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Novel Screening for Transthyretin Amyloidosis by Using Fat Ultrasonography

Yohei Misumi et al.

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Peripheral nerve ultrasonography in patients with transthyretin amyloidosis

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Extracellular volume quantification by dynamic equilibrium cardiac computed tomography in cardiac amyloidosis

Thomas A. Treibel et al.

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Transthyretin-derived amyloidosis: Probably a common cause of lumbar spinal stenosis

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Native T1 Mapping in Transthyretin Amyloidosis

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CMR-Based Differentiation of AL and ATTR Cardiac Amyloidosis

Jason N. Dungu et al.

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Spinal cord stimulation markedly ameliorated refractory neuropathic pain in transthyretin Val30Met familial amyloid polyneuropathy

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Diagnosis of sporadic transthyretin Val30Met familial amyloid polyneuropathy: a practical analysis

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Mass Spectrometric-Based Proteomic Analysis of Amyloid Neuropathy Type in Nerve Tissue

Christopher J. Klein et al.

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Transthyretin-related amyloidoses and the heart: a clinical overview

Claudio Rapezzi et al.

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Progression of transthyretin amyloid neuropathy after liver transplantation

Juris J. Liepnieks et al.

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The significance of carpal tunnel syndrome in transthyretin Val30Met familial amyloid polyneuropathy

Haruki Koike et al.

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Detection of left ventricular systolic dysfunction in cardiac amyloidosis with strain rate echocardiography

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Tissue damage in the amyloidoses: Transthyretin monomers and nonnative oligomers are the major cytotoxic species in tissue culture

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