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Advances in the treatment of hereditary transthyretin amyloidosis: A review

2019-09Journal article Open access
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dc.contributor.authorGertz, M.
dc.contributor.authorMauermann, M.
dc.contributor.authorGrogan, M.
dc.contributor.authorCoelho, T.
dc.date.accessioned2020-05-04T23:19:49Z
dc.date.available2020-05-04T23:19:49Z
dc.date.issued2019-09
dc.description.abstractIntroduction: 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. Methods: A PubMed search from 2008 to September 2018 was conducted to review the literature on ATTR. Results: 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 anti-inflammatory drug that is used off-label, are associated with side effects and/or unclear efficacy in certain patient populations. Thus, inotersen and patisiran are positioned to be the preferred therapeutic modalities. Conclusions: Important differences between inotersen and patisiran, including formulation, dosing, requirements for premedications, and safety monitoring, require an understanding and knowledge of each treatment for informed decision making.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationGertz MA, Mauermann ML, Grogan M, Coelho T. Advances in the treatment of hereditary transthyretin amyloidosis: A review. Brain Behav. 2019;9(9):e01371. doi:10.1002/brb3.1371pt_PT
dc.identifier.doi10.1002/brb3.1371pt_PT
dc.identifier.issn2162-3279
dc.identifier.issn2157-9032
dc.identifier.urihttp://hdl.handle.net/10400.16/2360
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherWiley Open Accesspt_PT
dc.relation.publisherversionhttps://onlinelibrary.wiley.com/doi/full/10.1002/brb3.1371pt_PT
dc.rights.urihttp://creativecommons.org/licenses/by-nc/4.0/pt_PT
dc.subjectamyloidpt_PT
dc.subjecthATTRpt_PT
dc.subjectinotersenpt_PT
dc.subjectpatisiranpt_PT
dc.subjecttransthyretin amyloidosispt_PT
dc.titleAdvances in the treatment of hereditary transthyretin amyloidosis: A reviewpt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.conferencePlaceUnited States of Americapt_PT
oaire.citation.issue9pt_PT
oaire.citation.startPagee01371pt_PT
oaire.citation.titleBrain and behaviorpt_PT
oaire.citation.volume9pt_PT
rcaap.rightsopenAccesspt_PT
rcaap.typearticlept_PT

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