臨床試驗
1.Klamerus KJ, Watsky E, Moller R, Wang R, Riley S. The effect of tafamidis on the QTc interval in healthy subjects. Br J Clin Pharmacol 2015;79(6):918-25.
2. Maurer MS, Grogan DR, Judge D, Mundayat R, Packman J, Lombardo I, Quyyumi A, Aarts J, Falk RH. Tafamidis in transthyretin amyloid cardiomyopathy: effects on transthyretin stabilization and clinical outcomes. Circ Heart Fail. 2015;8(3):519-26.
3.Sultan MB, Gundapaneni B, Schumacher J, Schwartz JH. Treatment with tafamidis slows disease progression in early-stage transthyretin cardiomyopathy. Clin Med Insights Cardiol. 2017;11:1-4.
4.Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M, Kristen AV, Grogan M, Witteles R, Damy T, Drachman BM, Shah SJ, Hanna M, Judge DP, Barsdorf AI, Huber P, Patterson TA, Riley S, Schumacher J, Stewart M, Sultan MB, Rapezzi C on behalf of the ATTR-ACT Study Investigators. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med. 2018;379:1007-1016.
5.Li B, Alvir J, Stewart M. Extrapolation of survival benefits in patients with transthyretin amyloid cardiomyopathy receiving tafamadis: analysis of the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial. Cardiol Ther. 2020;9:535-540.
6.Damy T, Garcia-Pavia P, Hanna M, Judge DP, Merlini G, Gundapaneni B, Patterson TA, Riley S, Schwartz JH, Sultan MB, Witteles R. Efficacy and safety of tafamidis doses in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and long-term extension study. Eur J Heart Fail. 2021;23(2):277-285.
7.Hanna M, Damy T, Grogan M, Stewart M, Gundapaneni B, Patterson TA, Schwartz JH, Sultan MB, Maurer MS. Impact of tafamidis on health-related quality of life in patients with transthyretin amyloid cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial). Am J Cardiol . 2021;141:98-105.
8.Rapezzi C, Elliott P, Damy T, Nativi-Nicolau J, Berk JL, Velazquez EJ, Boman K, Gundapaneni B, Patterson TA, Schwartz JH, Sultan MB, Maurer MS. Efficacy of tafamidis in patients with hereditary and wild-type transthyretin amyloid cardiomyopathy: further analyses from ATTR-ACT. JACC Heart Fail. JACC Heart Fail. 2021;9(2):115-123.
9.Miller AB, Januzzi JL, O’Neill BJ, Gundapaneni B, Patterson TA, Sultan MB, Lopez-Sendon J. Causes of cardiovascular hospitalization and death in patients with transthyretin amyloid cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial [ATTR-ACT]). Am J Cardiol. 2021;148:146-150.
10.Vong, C., Boucher, M., Riley, S. et al. Modeling of Survival and Frequency of Cardiovascular-Related Hospitalization in Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis. Am J Cardiovasc Drugs 2021; Online ahead of print.
11.Nativi-Nicolau J, Judge DP, Hoffman JE, Gundapaneni B, Keohane D, Sultan MB, Grogan M. Natural history and progression of transthyretin amyloid cardiomyopathy: insights from (ATTR-ACT). ESC Heart Failure. 2021; 8; 3875-3884
12.Elliott P, Drachman BM, Gottlieb SS, Hoffman JE, Hummel SL, Lenihan DJ, Ebede B, Gundapaneni B, Li B, Sultan MB, Shah SJ. Long-term survival with tafamidis in patients with transthyretin amyloid cardiomyopathy. Circ Heart Fail. 2021; 15; 1-11
真實世界數據
1.Planté-Bordeneuve V, Suhr O, Maurer M, White B, Grogan D, Coelho T. The Transthyretin Amyloidosis Outcomes Survey (THAOS) registry: design and methodology. Curr Med Res Opin. 2013;29(1):77-84.
2.Coelho T, Maurer MS, and Suhr OB, on behalf of the THAOS Investigators. THAOS – The Transthyretin Amyloidosis Outcomes Survey: Initial Report on Clinical Manifestations in Patients with Hereditary and Wild-Type Transthyretin Amyloidosis. Curr Med Res Opin. 2013;29(1):63-76.
3.Wixner J, Mundayat R, Karayal O, Anan I, Karling P, Suhr O, on behalf of the THAOS investigators. THAOS: gastrointestinal manifestations of transthyretin amyloidosis – common complications of a rare disease. Orphanet J Rare Dis. 2014;9:61.
4.Alvir J, Stewart M, Conceição I, on behalf of the THAOS Investigators. Latent class analysis to classify patients with transthyretin amyloidosis by signs and symptoms. Neurol Ther. 2015;1-14.
5.Damy T, Maurer MS, Rapezzi C, Planté-Bordeneuve V, Karayal ON, Mundayat R, Suhr OB, Kristen AV on behalf the THAOS investigators. Clinical, ECG and echocardiographic clues to the diagnosis of TTR-related cardiomyopathy: an insight through a worldwide registry – report from the Transthyretin Amyloidosis Outcome Survey (THAOS). Open Heart. 2016;3(1):e000289.
6.Maurer MS, Hanna M, Grogan M, Dispenzieri A, Witteles R, Drachman B, Judge DP, Lenihan DJ, Gottlieb SS, Shah SJ, Steidley E, Ventura H, Murali S, Silver MA, Jacoby D, Fedson S, Hummel SL, Kristen AV, Damy T, Planté-Bordeneuve V, Coelho T, Mundayat R, Suhr OB, Waddington Cruz M, Rapezzi C. Genotype and phenotype of transthyretin cardiac amyloidosis: THAOS (Transthyretin Amyloid Outcome Survey). J Am Coll Cardiol. 2016;68(2):161-172.
7.Maurer MS, Mundayat R, Rapezzi C. Reply: Val122Ile mt-ATTR has a worse survival than wt-ATTR cardiac amyloidosis. J Am Coll Cardiol. 2017;69(6):758-759.
8.Kristen AV, Maurer MS, Rapezzi C, Mundayat R, Suhr OB, Damy T, on behalf the THAOS investigators. Impact of genotype and phenotype on cardiac biomarkers in patients with transthyretin amyloidosis – report from the Transthyretin Amyloidosis Outcome Survey (THAOS). PLoS ONE. 2017;12(4):e0173086.
9.Mundayat R, Stewart M, Alvir J, Short S, Ong M-L, Keohane D, Rill D, Sultan MB. Positive effectiveness of tafamidis in delaying disease progression in transthyretin familial amyloid polyneuropathy up to 2 years: an analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Neurol Ther. 2018;7(1):87-101.
10.Rubin J, Steidley DE, Carlsson M, Ong M-L, Maurer MS. Myocardial contraction fraction by M-Mode echocardiography is superior to ejection fraction in predicting mortality in transthyretin amyloidosis. J Card Fail. 2018;Jul 12:S1071-9164(18)30258-6.
11.Waddington Cruz M, Vinicius Pinto M, Pinto LF, Gervais R, Dias M, Perez C, Mundayat R, Ong M-L, Pedrosa RC, Foguel D. Baseline Disease Characteristics in Brazilian Patients Enrolled in Transthyretin Amyloidosis Outcome Survey (THAOS). Arq Neuropsiquiatr. 2019;77(2):96-100.
12.Damy T, Kristen AV, Suhr OB, Maurer MS, Planté-Bordeneuve V, Yu CR, Ong ML, Coelho T, Rapezzi C; THAOS Investigators. Transthyretin cardiac amyloidosis in continental Western Europe: an insight through the Transthyretin Amyloidosis Outcomes Survey (THAOS). Eur Heart J. 2019 Apr 1. pii: ehz173. doi: 10.1093/eurheartj/ehz173. [Epub ahead of print].
13.Pinto MV, Pinto LF, Dias M, Santa Rosa RD, Mundayat R, Pedrosa RC, Waddington-Cruz M. Late-onset hereditary ATTR V30M amyloidosis with polyneuropathy: characterization of Brazilian subjects from the THAOS registry. Neurol Sci. 2019;May(403):1-6.
14.Huber P, Flynn A, Sultan MB, Li H, Rill D, Ebede B, Gundapaneni B, Schwartz JH. A comprehensive safety profile of tafamidis in patients with transthyretin amyloid polyneuropathy. Amyloid. 2019;26(4):203-209.
15.Sekijima Y, Mundayat R, Ishii T, Ando Y. The current status of the Transthyretin Amyloidosis Outcomes Survey (THAOS) in Japan. Amyloid. 2019;26(sup1):61-62.
16.Gonzalez-Duarte A, Barroso F, Mundayat R, Shapiro B. Blood pressure changes and orthostatic hypotension as measures of autonomic dysfunction in patients from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Auton Neurosci. 2019;222:102590.
17.Waddington-Cruz M, Ando Y, Amass L, Kiszko J, Chapman D, Sekijima Y . Feasibility of assessing progression of transthyretin amyloid polyneuropathy using nerve conduction studies: findings from the Transthyretin Amyloidosis Outcomes Survey (THAOS). J Peripher Nerv Syst. 2021;26(2):160-166.
18.Waddington-Cruz M, Wixner J, Amass L, Kiszko J, Chapman D, Ando Y. Characteristics of patients with late- versus early-onset Val30Met transthyretin amyloidosis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Neurol Ther. 2021; Online ahead of print.
19.Gentile L, Tournev I, Amass L, Chapman D, Mazzeo A. Phenotypic differences of Glu89Gln genotype in ATTR amyloidosis from endemic loci: data from Transthyretin Amyloidosis Outcomes Survey (THAOS). Cardiol Ther. 2021; 10; 481-490
20.Gonzalez-Moreno J, Losada-Lopez I, Cisneros-Barroso E, Garcia-Pavia P, Gonzalez-Costello J, Munoz-Beamud F, Campistol JM, Torron RF, Chapman D, Amass L. A descriptive analysis of ATTR amyloidosis in Spain from the Transthyretin Amyloidosis Outcomes Survey. Neurol Ther. 2021; 10: 833-845
21.Caponetti G, Rapezzi C, Gagliardi C, Milandri A, Dispenzieri A, Kristen A, Wixner J, Maurer M, Garcia-Pavia P, Tournev I, Plante-Bordeneuve V, Chapman D, Amass L. Sex-related risk of cardiac involvement in hereditary ATTR amyloidosis: insights from THAOS. JACC Heart Fail. 2021; 10; 71-72
22.Nativi-Nicolau J, Siu A, Dispenzieri A, Maurer MS, Rapezzi C, Kristen A, Garcia-Pavia P, LoRusso S, Waddington-Cruz M, Lairez O, Witteles R, Chapman D, Amass L, Grogan M. Temporal trends of wild-type transthyretin amyloid cardiomyopathy in the Transthyretin Amyloidosis Outcomes Survey. JACC CardioOncol. 2021;3(4):537-46; 4; 537-546
23.Coelho T, Dispenzieri A, Grogan M, Conceição I, Waddington-Cruz M, Kristen AV, Wixner J, Diemberger I, Gonzalez-Moreno J, Maurer MS, Plante-Bordeneuve V, Garcia Pavia P, Tournev I, Gonzalez-Costello J, Cariou E, González-Duarte A, Glass O, Chapman D, Amass L. Patients with transthyretin amyloidosis enrolled in THAOS between 2018 and 2021 continue to experience substantial diagnostic delay. Amyloid. 2023;30:445–448. doi: 10.1080/13506129.2023.2229484
24.Gentile L, Coelho T, Dispenzieri A, Conceicao I, Waddington-Cruz M, Kristen A, Wixner J, Diemberger I, Gonzalez Moreno J, Cariou E, Maurer M, Plante Bordeneuve V, Garcia Pavia P, Tournev I, Gonzalez Costello J, Gonzalez Duarte A, Grogan M, Mazzeo A, Chapman D, Gupta P, Glass O, Amass L. A 15-year consolidated overview of data in over 6000 patients from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Orphanet Journal of Rare Diseases. 2023;18:350. doi: 10.1186/s13023-023-02962-5
25. González-Moreno J, Dispenzieri A, Grogan M, Coelho T, Tournev I, Waddington-Cruz M, Wixner J, Diemberger I, Garcia-Pavia P, Chapman D, Gupta P, Glass O, Amass L. Clinical and genotype characteristics and symptom migration of patients with mixed phenotype transthyretin amyloidosis from the Transthyretin Amyloidosis Outcomes Survey. Cardiology and Therapy. 2024;13(1):117-135. doi: 10.1007/s40119-023-00344-3.
26. Gentile L, Diemberger I, Plante-Bordeneuve V, Mazzeo A, Dori A, Luigetti M, DiPaolantonio A, Dispenzieri A, Grogan M, Waddington-Cruz M, Adams D, Inamo J, Kristen A, Lino Cirami C, Chapman D, Gupta P, Glass O, Amass L. Phenotypic characteristics of Phe64Leu, Ile68Leu, Ile107Val and Ser77Tyr ATTRv genotypes from the Transthyretin Amyloidosis Outcomes Survey (THAOS). PLoS One. 2024;19(1):e0292435. doi: 10.1371/journal.pone.0292435
27. Mora N, Dispenzieri A, Kristen A, Maurer M, Diemberger I, Drachman B, Grogan M, Gupta P, Glass O, Amass L, Garcia-Pavia P. Age- and Sex-Related Differences in Patients with Wild-Type Transthyretin Amyloidosis: Insights from THAOS. JACC Advances. 2024;3(8):101086. doi: 10.1016/j.jacadv.2024.101086.
28. Garcia-Pavia P, Kristen A, Drachman B, Carlsson M, Amass L, Angeli F, Maurer M. Survival in a Real-World Cohort of Patients With Transthyretin Amyloid Cardiomyopathy Treated With Tafamidis: An Analysis From the Transthyretin Amyloidosis Outcomes Survey (THAOS). Journal of Cardiac Failure. 2024. doi: 10.1016/j.cardfail.2024.06.003