Prediction of long-term survival after liver transplantation for familial transthyretin amyloidosis.
نویسندگان
چکیده
Spanish Ministry of Economy and Competitiveness (grant N BFU 2012-36241) and Programa INNPACTO (grant N IPT-2011-0817-010000) (to Dr. Martin), and from the Agence Nationale de la Recherche (Programme Blanc BCNCT) (to Dr. Lambert). Dr. Alves was a recipient of a PhD grant SFRH/BD/27990/2006 and research grant PTDC/SAU-GMG/101874/2008 from FCT. Mrs. Medeiros was supported by a research grant from the National Institute of Health Doutor Ricardo Jorge (BRJ-DPS/2012). Mr. Benito-Vicente was supported by a grant PIF (2014/2015) Gobierno Vasco. Alirocumab was obtained from Sanofi-Regeneron. Dr. Lambert has received honoraria from Sanofi-Regeneron, Amgen, and Pfizer and research funding from Sanofi-Regeneron. All other authors have reported that they have no relationships relevant to the contents of this paper to disclose. Drs. Alves and Etxebarria contributed equally to this work.
منابع مشابه
Impact of liver transplantation on transthyretin-related ocular amyloidosis in Japanese patients.
OBJECTIVE To evaluate the long-term impact of liver transplantation on ocular manifestations of familial amyloid polyneuropathy (FAP) in Japanese patients. METHODS Medical records were retrospectively reviewed in a long-term follow-up study. Of 52 patients with FAP amyloidogenic transthyretin Val30Met, 22 patients underwent liver transplantation. We assessed ocular manifestations, including a...
متن کاملLiver transplantation and transthyretin amyloidosis.
Liver transplantation as a specific treatment of transthyretin amyloidosis was first performed in 1990. The rationale for this treatment was that removal of the source (liver) of the amyloid precursor protein (mutated transthyretin) would stop progression of the disease. Indeed, after orthotopic liver transplantation (OLT), mutant transthyretin (TTR) is rapidly cleared from circulation. In the ...
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progress in Kumamoto, Japan (1967–2010). Proc Jap Acad Ser B Phys Biol Sci Nippon Gakushiin 2010;86:694–706. 4. Rapezzi C, Quarta CC, Obici L, Perfetto F, Longhi S, Salvi F, Biagini E, Lorenzini M, Grigioni F, Leone O, Cappelli F, Palladini G, Rimessi P, Ferlini A, Arpesella G, Pinna AD, Merlini G, Perlini S. Disease profile and differential diagnosis of hereditary transthyretin-related amyloid...
متن کاملATTR-FAP: liver transplantation vs oral medication, how and when
Background Liver transplantation (LTx), introduced in 1990, has served as the only available treatment with capacity to halt the progress of disease in transthyretin amyloidosis. For the most common variant, Val30Met, the effect of a new liver is well known leading to stabilization in the majority of patients. However, not all patients are helped by transplantation. Progress of cardiac amyloido...
متن کاملA Heart too Stiff to Beat: A Case of Familial Transthyretin Amyloidosis Cardiomyopathy
Heart failure is a common clinical syndrome caused by a variety of cardiac diseases. We report a rare case of familial transthyretin amyloidosis cardiomyopathy to heighten the awareness of this rare but lethal cause of heart failure, as therapeutic interventions such as liver or heart transplant could be curative in selected patients.
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ورودعنوان ژورنال:
- Journal of the American College of Cardiology
دوره 66 19 شماره
صفحات -
تاریخ انتشار 2015