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      Monitoring treatment response to tafamidis by serial native T1 and extracellular volume in transthyretin amyloid cardiomyopathy

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          Abstract

          Tafamidis meglumine, a transthyretin (TTR) stabilizer, is effective in delaying the progression of neuropathy in TTR amyloidosis with Val30Met mutations. However, its efficacy in TTR amyloid cardiomyopathy is not fully elucidated. Herein, we report a 73‐year‐old Japanese man with a diagnosis of TTR amyloid cardiomyopathy with Val30Met mutation treated with tafamidis. To evaluate treatment response, cardiac magnetic resonance imaging was performed before and after 12 months of tafamidis treatment. Native T1, extracellular volume, and left ventricular mass showed no obvious worsening, and findings of other diagnostic studies also supported the efficacy of tafamidis to delay the progression of amyloid cardiomyopathy. Our case suggests that serial native T1 and extracellular volume may be novel non‐invasive imaging methods to monitor the treatment response to TTR stabilizers in cardiac amyloidosis and also that tafamidis may be effective in suppressing cardiac progression in TTR amyloid cardiomyopathy with Val30Met mutation.

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          TTR (Transthyretin) Stabilizers Are Associated With Improved Survival in Patients With TTR Cardiac Amyloidosis.

          TTR (transthyretin) cardiac amyloidosis is caused by dissociation of TTR into monomers, which misassemble into amyloid fibrils. TTR stabilizers act at the dimer-dimer interface to prevent dissociation. We investigated differences in survival among patients with TTR cardiac amyloidosis on stabilizer medications compared with those not on stabilizers.
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            Distinct characteristics of amyloid deposits in early- and late-onset transthyretin Val30Met familial amyloid polyneuropathy.

            Late-onset transthyretin Val30Met-associated familial amyloid polyneuropathy (FAP ATTR Val30Met) cases unrelated to endemic foci in Japan show different clinicopathological features from the conventional early-onset cases in endemic foci. We compared the characteristics of amyloid deposits in early-onset FAP ATTR Val30Met cases in endemic foci and late-onset cases in non-endemic areas. Amyloid deposits in three early-onset cases from endemic foci and five late-onset cases from non-endemic areas were systematically examined post-mortem. Amyloid deposits in early-onset cases were highly congophilic and showed strong apple-green birefringence with Congo red staining and had long, parallel fibrils in most organs. On the other hand, those in late-onset cases were generally weakly congophilic and showed faint apple-green birefringence with Congo red staining and had short, haphazard fibrils. In the renal glomus and adrenal gland of early-onset cases, the characteristics of amyloid deposits were similar to those observed in late-onset cases. Analysis of cardiac amyloid using surface enhanced desorption/ionization time-of-flight mass spectrometry indicated that most transthyretin (TTR) was variant in early-onset cases, while more than half was composed of wild-type TTR in late-onset cases. Although characteristics of amyloid deposits may differ among individual organs of respective cases, especially in early-onset cases, the pattern was distinct between early- and late-onset cases. Amyloid deposition in late-onset cases may be similar to that observed in senile systemic amyloidosis with wild-type TTR deposition, suggesting that aging may play an important role in these cases.
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              Recent advances in diagnosis and treatment of cardiac amyloidosis.

              Cardiac amyloidosis (CA) has been believed to be a rare disease for a long time, but recent sophisticated diagnostic modalities demonstrate that a considerable number of CA patients are hidden among those diagnosed with heart failure. Prognosis of CA was poor, but recent developments in therapeutic interventions have improved survival in these patients. Therefore, early detection and precise diagnosis is clinically important. In this review article, we overview recent progress in diagnosis and treatment for CA.
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                Author and article information

                Contributors
                okada.atsushi.hp@ncvc.go.jp
                Journal
                ESC Heart Fail
                ESC Heart Fail
                10.1002/(ISSN)2055-5822
                EHF2
                ESC Heart Failure
                John Wiley and Sons Inc. (Hoboken )
                2055-5822
                27 November 2018
                February 2019
                : 6
                : 1 ( doiID: 10.1002/ehf2.v6.1 )
                : 232-236
                Affiliations
                [ 1 ] Department of Cardiovascular Medicine National Cerebral and Cardiovascular Center Osaka Japan
                [ 2 ] Department of Radiology National Cerebral and Cardiovascular Center Osaka Japan
                [ 3 ] Department of Pathology National Cerebral and Cardiovascular Center Osaka Japan
                [ 4 ] Department of Hematology Japan Community Health care Organization Kyoto Kuramaguchi Medical Center Kyoto Japan
                [ 5 ] Department of Medicine (Neurology and Rheumatology) Shinshu University School of Medicine Matsumoto Japan
                [ 6 ] Department of Biological Sciences for Intractable Neurological Diseases, Institute for Biomedical Sciences Shinshu University Matsumoto Japan
                Author notes
                [*] [* ] Correspondence to: Atsushi Okada, Department of Cardiovascular Medicine, National Cerebral and Cardiovascular Center, 5‐7‐1 Fujishiro‐dai, Suita, Osaka 565‐8565, Japan. Tel: +81 6 6833 5012; Fax: +81 6 6872 7486. Email: okada.atsushi.hp@ 123456ncvc.go.jp

                Article
                EHF212382 ESCHF-18-00163
                10.1002/ehf2.12382
                6352892
                30478886
                e7ce1980-210f-4455-be95-95a7d527b3db
                © 2018 The Authors. ESC Heart Failure published by John Wiley & Sons Ltd on behalf of the European Society of Cardiology.

                This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

                History
                : 09 August 2018
                : 13 October 2018
                Page count
                Figures: 3, Tables: 0, Pages: 5, Words: 1390
                Categories
                Case Report
                Case Reports
                Custom metadata
                2.0
                ehf212382
                February 2019
                Converter:WILEY_ML3GV2_TO_NLMPMC version:5.5.7 mode:remove_FC converted:30.01.2019

                transthyretin,amyloidosis,tafamidis,cardiac magnetic resonance,native t1,extracellular volume

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