Revisión anual
Tratamiento de la amiloidosis cardÃÂaca por transtiretina
Federico Weckesser, Juan P Costabel
Revista del Consejo Argentino de Residentes de CardiologÃÂa ;():0161-0167
La amiloidosis representa un conjunto de enfermedades ocasionadas por el depósito de un grupo de proteínas denominadas “amiloide”.
El compromiso cardíaco es la principal causa de morbimortalidad en la amiloidosis sistémica, independientemente de la patogenia productora del amiloide subyacente.
Solo dos tipos de amiloidosis son las responsables de más del 95% de las amiloidosis cardíacas: la amiloidosis por cadenas livianas y la amiloidosis por transtiretina (ATTR).
En el siguiente trabajo nos propusimos resumir los conceptos más importantes en relación al manejo de los pacientes con ATTR.
Palabras clave: amiloidosis, tratamiento, amiloidosis de cadenas livianas,
Amyloidosis represents a group of diseases caused by the deposit of a group of proteins called “amyloid”.
Cardiac involvement is the main cause of morbidity and mortality in systemic amyloidosis, regardless of the underlying amyloid-producing pathogenesis.
Only two types of amyloidosis are responsible for more than 95% of cardiac amyloidosis: light chain amyloidosis and transthyretin amyloidosis (ATTR).
In the following study, we set out to summarize the most important concepts in relation to the management of patients with ATTR.
Keywords: amyloidosis, treatment, immunoglobulin light-chain amyloidosis,
Los autores declaran no poseer conflictos de intereses.
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Recibido 2020-06-12 | Aceptado 2020-07-19 | Publicado 2020-08-31
Manolis AS, Manolis AA, Manolis TA, Melita H. Cardiac amyloidosis: An underdiagnosed/underappreciated disease. Eur J Intern Med 2019;67:1-13.
Li L, Duan XJ, Sun Y, Lu Y, Xu HY, Wang QZ, et al. [Classification of cardiac amyloidosis: an immunohistochemical analysis]. Zhonghua Bing Li Xue Za Zhi 2018;47(2):105-9.
Ihne S, Morbach C, Obici L, Palladini G, Störk S. Amyloidosis in Heart Failure. Curr Heart Fail Rep. 2019;16(6):285-303.
Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M, et al. Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy. N Engl J Med 2018;379(11):1007-16.
Paton DM. Tafamidis: a selective transthyretin stabilizer to treat wild-type ATTR amyloidosis and hereditary ATTR amyloidosis with cardiomyopathy. Drugs Today (Barc) 2019;55(12):727-34.
Wixner J, Westermark P, Ihse E, Pilebro B, Lundgren HE, Anan I. The Swedish open-label diflunisal trial (DFNS01) on hereditary transthyretin amyloidosis and the impact of amyloid fibril composition. Amyloid 2019;26(sup1):39-40.
Berk JL, Suhr OB, Obici L, Sekijima Y, Zeldenrust SR, Yamashita T, et al. Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial. JAMA 2013;310(24):2658-67.
Ikram A, Donnelly JP, Sperry BW, Samaras C, Valent J, Hanna M. Diflunisal tolerability in transthyretin cardiac amyloidosis: a single center’s experience. Amyloid 2018;25(3):197-202.
Lohrmann G, Pipilas A, Mussinelli R, Gopal DM, Berk JL, Connors LH, et al. Stabilization of Cardiac Function With Diflunisal in Transthyretin (ATTR) Cardiac Amyloidosis. J Card Fail 2019;S1071-9164(19)31435-6.
Torres-Arancivia C, Connors LH. Effect of diflunisal on clusterin levels in ATTRwt amyloidosis. Amyloid. 2019;26(sup1):49-50.
Rezai-Zadeh K, Shytle D, Sun N, Mori T, Hou H, Jeanniton D, et al. Green tea epigallocatechin-3-gallate (EGCG) modulates amyloid precursor protein cleavage and reduces cerebral amyloidosis in Alzheimer transgenic mice. J Neurosci. 2005;25(38):8807-8814.
Mereles D, Buss SJ, Hardt SE, Hunstein W, Katus HA. Effects of the main green tea polyphenol epigallocatechin-3-gallate on cardiac involvement in patients with AL amyloidosis. Clin Res Cardiol. 2010;99(8):483–90.
Kristen A V, Lehrke S, Buss S, Mereles D, Steen H, Ehlermann P, et al.Green tea halts progression of cardiac transthyretin amyloidosis: an observational report. Clin Res Cardiol. 2012;101(10):805-813.
Aus dem Siepen F, Bauer R, Aurich M, Buss SJ, Steen H, Altland K, et al. Green tea extract as a treatment for patients with wild-type transthyretin amyloidosis: an observational study. Drug Des Devel Ther. 2015;(9):6319-6325.
Saeki K, Hayakawa S, Nakano S, Ito S, Oishi Y, Suzuki Y, et al. In Vitro and In Silico Studies of the Molecular Interactions of Epigallocatechin-3-O-gallate (EGCG) with Proteins That Explain the Health Benefits of Green Tea. Molecules. 2018;23(6):1295. 16.
Judge DP, Heitner SB, Falk RH, Maurer MS, Shah SJ, Witteles RM, et al.Transthyretin Stabilization by AG10 in Symptomatic Transthyretin Amyloid Cardiomyopathy. JACC. 2019;74(3):285-295.
Penchala SC, Connelly S, Wang Y, Park MS, Zhao L, Baranczak A, et al.AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretin. Proc Natl Acad Sci U S A. 2013;110(24):9992-9997.
Fox JC, Hellawell JL, Rao S, O’Reilly T, Lumpkin R, Jernelius J, et al. First-in-Human Study of AG10, a Novel, Oral, Specific, Selective, and Potent Transthyretin Stabilizer for the Treatment of Transthyretin Amyloidosis: A Phase 1 Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Adult Volunteers. Clin Pharmacol Drug Dev. 2020;9(1):115-129.
Miller M, Pal A, Albusairi W, Joo H, Pappas B, Haque Tuhin MT, et al. Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis. J Med Chem 2018;61(17):7862-7876.
Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, et al. Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018;379(1):22-31.
Dasgupta NR, Rissing SM, Smith J, Jung J, Benson MD. Inotersen therapy of transthyretin amyloid cardiomyopathy. Amyloid. 2020;27(1):52-58.
Adams D, Gonzalez-Duarte A, O’Riordan WD, Yang C-C, Ueda M, Kristen A V, et al. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018;379(1):11-21.
Zhang X, Goel V, Attarwala H, Sweetser MT, Clausen VA, Robbie GJ. Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure-Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin-Mediated (hATTR) Amyloidosis. J Clin Pharmacol. 2020;60(1):37-49.
Planté-Bordeneuve V, Lin H, Gollob J, Agarwal S, Betts M, Fahrbach K, et al. An indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy. Expert Opin Pharmacother. 2019;20(4):473-481.
Solomon SD, Adams D, Kristen A, Grogan M, González-Duarte A, Maurer MS, et al. Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis. Circulation. 2019;139(4):431-443.
Adams D, Gonzalez-Duarte A, O’Riordan WD, Yang C-C, Ueda M, Kristen A V, et al. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018;379(1):11-21.
Yang J. Patisiran for the treatment of hereditary transthyretin-mediated amyloidosis. Expert Rev Clin Pharmacol. 2019;12(2):95-99.
Kristen AV, Ajroud-Driss S, Conceição I, Gorevic P, Kyriakides T, Obici L. Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis. Neurodegener Dis Manag. 2019;9(1):5-23.
Hoy SM. Patisiran: First Global Approval. Drugs. 2018;78(15):1625-1631.
Habtemariam BA, Karsten V, Attarwala H, Goel V, Melch M, Clausen VA, et al.Single-Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N-acetylgalactosamine-Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects. Clin Pharmacol Ther. 2020;10.1002/cpt.1974.
Piccoli GB, Hachemi M, Molfino I, Coindre JP, Boursot C. Doxycycline treatment in dialysis related amyloidosis: discrepancy between antalgic effect and inflammation, studied with FDG-positron emission tomography: a case report. BMC Nephrol. 2017;18(1):285.
Karlstedt E, Jimenez-Zepeda V, Howlett JG, White JA, Fine NM. Clinical Experience With the Use of Doxycycline and Ursodeoxycholic Acid for the Treatment of Transthyretin Cardiac Amyloidosis. J Card Fail. 2019;25(3):147-153.
D’Souza A, Flynn K, Chhabra S, Dhakal B, Hamadani M, Jacobsen K,et al. Rationale and design of DUAL study: Doxycycline to Upgrade response in light chain (AL) amyloidosis (DUAL): A phase 2 pilot study of a two-pronged approach of prolonged doxycycline with plasma cell-directed therapy in the treatment of AL amyloidosis. Contemp Clin Trials Commun. 2017; 24(8):33-38.
Witteles RM. Doxycycline and Ursodiol for ATTR Amyloidosis: Not Ready for Prime Time. J Card Fail. 2019;25(3):154-155.
Wechalekar AD, Whelan C. Encouraging impact of doxycycline on early mortality in cardiac light chain (AL) amyloidosis. Blood Cancer J. 2017;7(3):e546.
Castaño A, Drachman BM, Judge D, Maurer MS. Natural history and therapy of TTR-cardiac amyloidosis: emerging disease-modifying therapies from organ transplantation to stabilizer and silencer drugs. Heart Fail Rev. 2015;20(2):163-178.
Hawkins PN, Ando Y, Dispenzeri A, Gonzalez-Duarte A, Adams D, Suhr OB. Evolving landscape in the management of transthyretin amyloidosis. Ann Med. 2015;47(8):625-638.
Bodin K, Ellmerich S, Kahan MC, Tennent GA, Loesch A, Gilbertson JA, et al. Antibodies to human serum amyloid P component eliminate visceral amyloid deposits. Nature. 2010;468(7320):93-97.
Balciunaite G, Rimkus A, Zurauskas E, Zaremba T, Palionis D, Valeviciene N, et al. Transthyretin cardiac amyloidosis in aortic stenosis: Prevalence, diagnostic challenges, and clinical implications. Hellenic J Cardiol. 2019;61(2):92-98.
Java AP, Greason KL, Dispenzieri A, Grogan M, Maleszewki JJ, Daly RC, et al. Cardiac amyloidosis in aortic stenosis-icebergs and Archimedes’ principle. J Thorac Cardiovasc Surg. 2018;156(3):966-967.
Penalver J, Ambrosino M, Jeon HD, Agrawal A, Kanjanahattakij N, Pitteloud M, et al. Transthyretin Cardiac Amyloidosis and Aortic Stenosis: Connection and Therapeutic Implications. Curr Cardiol Rev. 2020;16(3):221-230.
Ternacle J, Krapf L, Mohty D, Magne J, Nguyen A, Galat A, et al. Aortic Stenosis and Cardiac Amyloidosis: JACC. 2019;74(21):2638-2651.
Peskó G, Jenei Z, Varga G, Apor A, Vágó H, Czibor S, et al. Coexistence of aortic valve stenosis and cardiac amyloidosis: echocardiographic and clinical significance. Cardiovasc Ultrasound. 2019;17(1):32.
Çiçek S. Cardiac amyloidosis and aortic stenosis: Great masquerader back again!. J Thorac Cardiovasc Surg. 2018;156(1):104-105.
Scully PR, Moon JC, Treibel TA. Cardiac amyloidosis in aortic stenosis: The tip of the iceberg. J Thorac Cardiovasc Surg. 2018;156(3):965-966.
Giancaterino S, Urey MA, Darden D, Hsu JC. Management of Arrhythmias in Cardiac Amyloidosis. JACC Clin Electrophysiol. 2020;6(4):351-361.
John RM. Arrhythmias in Cardiac Amyloidosis. J Innov Card Rhythm Manag. 2018;9(3):3051-3057.
Mohty D, Damy T, Cosnay P, EchaHidi N, Casset-senon D, Virot P, et al. Cardiac amyloidosis: updates in diagnosis and management. Arch Cardiovasc Dis. 2013;106(10):528-540.
Cheung CC, Roston TM, Andrade JG, Bennett MT, Davis MK. Arrhythmias in Cardiac Amyloidosis: Challenges in Risk Stratification and Treatment. Can J Cardiol. 2020;36(3):416-423.
Hamon D, Algalarrondo V, Gandjbakhch E, Extramiana F, Marijon E, Elbaz N, et al. Outcome and incidence of appropriate implantable cardioverter-defibrillator therapy in patients with cardiac amyloidosis. Int J Cardiol. 2016;222:562-568.
John RM, Stern DL. Use of Implantable Electronic Devices in Patients With Cardiac Amyloidosis. Can J Cardiol. 2020;36(3):408-415.
Varr BC, Zarafshar S, Coakley T, Liedtke M, Lafayette RA, Arai S, et al.Implantable cardioverter-defibrillator placement in patients with cardiac amyloidosis. Heart Rhythm. 2014;11(1):158-162.
El-Am EA, Dispenzieri A, Melduni RM, Ammash NM, White RD, Hodge DO, et al. Direct Current Cardioversion of Atrial Arrhythmias in Adults With Cardiac Amyloidosis. JACC. 2019;73(5):589-597.
Manolis AS, Manolis AA, Manolis TA, Melita H. Cardiac amyloidosis: An underdiagnosed/underappreciated disease. Eur J Intern Med. 2019;67:1-13.
Ballantyne B, Manian U, Sheyin O, Davey R, De S. Stroke risk and atrial mechanical dysfunction in cardiac amyloidosis. ESC Heart Fail. 2020;7(2):705-707.
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Consejo Argentino de Residentes de CardiologÃÂa
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Etiquetas
amiloidosis, tratamiento, amiloidosis de cadenas livianas,
Tags
amyloidosis, treatment, immunoglobulin light-chain amyloidosis,
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