Advances in the treatment of hereditary transthyretin amyloidosis: A review

Brain and Behavior
Morie A GertzTeresa Coelho

Abstract

Amyloid transthyretin amyloidosis (ATTR) is a progressive and often fatal disease caused by the buildup of mutated (hereditary ATTR [hATTR]; also known as ATTR variant [ATTRv]) or normal transthyretin (wild-type ATTR) throughout the body. Two new therapies-inotersen, an antisense oligonucleotide therapy, and patisiran, an RNA interference therapy-received marketing authorization and represent a significant advance in the treatment of amyloidosis. Herein, we describe the clinical presentation of ATTR, commonly used procedures in its diagnosis, and current treatment landscape for ATTR, with a focus on hATTR. A PubMed search from 2008 to September 2018 was conducted to review the literature on ATTR. Until recently, there have been few treatment options for polyneuropathy of hATTR. Inotersen and patisiran substantially reduce the amyloidogenic precursor protein transthyretin and have demonstrated efficacy in patients with early- and late-stage disease and in slowing or improving neuropathy progression. In contrast, established therapies, such as liver transplantation, typically reserved for patients with early-stage disease, and tafamidis, indicated for the treatment of early-stage disease in Europe, or diflunisal, a nonsteroidal a...Continue Reading

References

Nov 17, 2006·Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis·Yoshiki SekijimaJeffery W Kelly
Dec 5, 2008·Journal of the American Geriatrics Society·Matthew S Robbins, Jay Yasen
May 10, 2011·The American Journal of Cardiology·Merrill D BensonJohn C Kincaid
Jun 23, 2011·Journal of the Peripheral Nervous System : JPNS·Manuel CappellariGian Maria Fabrizi
May 31, 2012·Proceedings of the National Academy of Sciences of the United States of America·Christine E BulawaRichard Labaudinière
Sep 6, 2012·Circulation·Frederick L Ruberg, John L Berk
May 30, 2013·Proceedings of the National Academy of Sciences of the United States of America·Sravan C PenchalaMamoun M Alhamadsheh
Jul 10, 2013·European Journal of Neurology : the Official Journal of the European Federation of Neurological Societies·P LozeronUNKNOWN French Network for FAP (CORNAMYL)
Oct 9, 2013·Journal of Cardiovascular Translational Research·Giampaolo MerliniDonna R Grogan
Dec 26, 2013·JAMA : the Journal of the American Medical Association·John L BerkUNKNOWN Diflunisal Trial Consortium
Dec 9, 2014·Liver Transplantation : Official Publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society·Andreia CarvalhoLuísa Lobato
Apr 19, 2015·Health and Quality of Life Outcomes·Juntana Pattanaphesaj, Montarat Thavorncharoensap
May 29, 2015·Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis·Yoshiki SekijimaShu-ichi Ikeda
Aug 6, 2015·Journal of Neurology, Neurosurgery, and Psychiatry·A S CarrM M Reilly
Sep 5, 2015·Orphanet Journal of Rare Diseases·Ole B SuhrDavid Adams
Oct 11, 2015·Journal of Nuclear Cardiology : Official Publication of the American Society of Nuclear Cardiology·Adam CastañoSabahat Bokhari
Nov 28, 2015·Journal of the American College of Cardiology·Morie A GertzGiampaolo Merlini
Dec 15, 2015·Journal of the Peripheral Nervous System : JPNS·Isabel ConceiçãoLeslie Amass
Jan 15, 2016·Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis·Kosuke OkumuraYukio Ando
Feb 9, 2016·Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis·Michitaka NakagawaShu-Ichi Ikeda
Feb 26, 2016·European Heart Journal·Arnault GalatThibaud Damy
Mar 17, 2016·Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis·Jeffrey N HigakiGene G Kinney
May 5, 2016·Circulation·Julian D GillmorePhilip N Hawkins
Oct 21, 2016·Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis·Elizabeth J AckermannBrett P Monia
Jul 9, 2016·Journal of the American College of Cardiology·Mathew S MaurerUNKNOWN THAOS Investigators
Jul 17, 2016·Muscle & Nerve·Teresa CoelhoDonna R Grogan
Sep 2, 2016·Journal of Nuclear Cardiology : Official Publication of the American Society of Nuclear Cardiology·Sabahat BokhariMathew S Maurer
Apr 25, 2017·Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis·Sebastián Ernesto AzorínJosep Maria Campistol
Jul 14, 2017·PloS One·Tadeo E SaldañoSebastian Fernandez-Alberti
Aug 2, 2017·Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis·Fabio A BarrosoMarla B Sultan
Sep 13, 2017·JAMA : the Journal of the American Medical Association·Hallie I GellerRodney H Falk
Dec 1, 2017·Progress in Transplantation : Official Publication, North American Transplant Coordinators Organization ... [et Al.]·Dipti BanerjeeAngela Dispenzieri
Dec 20, 2017·Cleveland Clinic Journal of Medicine·Joseph P Donnelly, Mazen Hanna

❮ Previous
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Citations

Jul 2, 2020·Mass Spectrometry Reviews·Jacob W McCabeDavid H Russell
Jul 8, 2020·Chemical Society Reviews·Pu Chun KeRaffaele Mezzenga
Jun 5, 2020·The Canadian Journal of Neurological Sciences. Le Journal Canadien Des Sciences Neurologiques·Dina NamiranianRami Massie
Jul 30, 2020·Clinical Cardiology·Catherine TengQi Liu
Nov 8, 2019·Archives of Pathology & Laboratory Medicine·Gabriel Giannini, Cynthia C Nast
Sep 16, 2020·Journal of Internal Medicine·E MuchtarM A Gertz
Jan 16, 2020·Biomedicines·Andrei A DeviatkinAlexander N Lukashev
Oct 10, 2020·Annual Review of Pharmacology and Toxicology·C Frank BennettDon W Cleveland
Aug 31, 2020·Mayo Clinic Proceedings·Surendra DasariEllen D McPhail
Aug 14, 2020·Bioorganic Chemistry·Valentina LoconteRodolfo Berni
Jan 27, 2021·International Journal of Environmental Research and Public Health·Elena García-GarcíaMarlon Félix Cárdenas Bonnet
Dec 9, 2020·Molecular Therapy : the Journal of the American Society of Gene Therapy·Yutaro AsamiTakanori Yokota
Jan 21, 2021·Journal of Personalized Medicine·Emily R SoperNoura S Abul-Husn
Dec 29, 2020·Frontiers in Molecular Neuroscience·Filipa BezerraMaria Rosário Almeida
Feb 16, 2021·The Journal of Clinical Investigation·Michael Camilleri
Feb 13, 2021·Diagnostics·Magdalena KramerSophia Willikens
Feb 26, 2021·The Canadian Journal of Neurological Sciences. Le Journal Canadien Des Sciences Neurologiques·Monica AlcantaraVera Bril
May 1, 2021·Brain Sciences·Luca GentileAnna Mazzeo
May 23, 2021·Clinics in Laboratory Medicine·David L Murray, Surendra Dasari
Aug 8, 2021·Genetics in Medicine : Official Journal of the American College of Medical Genetics·David R MurdockRichard A Gibbs
Aug 17, 2021·Cardiology in Review·Matthew Capustin, William H Frishman
May 12, 2020·Acta Haematologica·Maria M Picken

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Methods Mentioned

BETA
electrophoresis
antisense oligonucleotide
gene knockdown

Clinical Trials Mentioned

NCT00409175
NCT00791492
NCT00925002
NCT01994889
NCT00294671
NCT01737398
NCT02175004
NCT01960348
NCT02510261
NCT03336580

Software Mentioned

ApotheCom
APOLLO

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