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Predictive model of response to tafamidis in hereditary ATTR polyneuropathy

2019-06-20Journal article Open access
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dc.contributor.authorMonteiro, C.
dc.contributor.authorMesgazardeh, J.
dc.contributor.authorAnselmo, J.
dc.contributor.authorFernandes, J.
dc.contributor.authorNovais, M.
dc.contributor.authorRodrigues, C.
dc.contributor.authorBrighty, G.
dc.contributor.authorPowers, D.
dc.contributor.authorPowers, E.
dc.contributor.authorCoelho, T.
dc.contributor.authorKelly, J.
dc.date.accessioned2020-05-22T17:43:00Z
dc.date.available2020-05-22T17:43:00Z
dc.date.issued2019-06-20
dc.description.abstractBACKGROUNDThe hereditary transthyretin (TTR) amyloidoses are a group of diseases for which several disease-modifying treatments are now available. Long-term effectiveness of these therapies is not yet fully known. Moreover, the existence of alternative therapies has resulted in an urgent need to identify patient characteristics that predict response to each therapy.METHODSWe carried out a retrospective cohort study of 210 patients with hereditary TTR amyloidosis treated with the kinetic stabilizer tafamidis (20 mg qd). These patients were followed for a period of 18-66 months, after which they were classified by an expert as responders, partial responders, or nonresponders. Correlations between baseline demographic and clinical characteristics, as well as plasma biomarkers and response to therapy, were investigated.RESULTS34% of patients exhibited an almost complete arrest of disease progression (classified by an expert as responders); 36% had a partial to complete arrest in progression of some but not all disease components (partial responders); whereas the remaining 30% continued progressing despite therapy (nonresponders). We determined that disease severity, sex, and native TTR concentration at the outset of treatment were the most relevant predictors of response to tafamidis. Plasma tafamidis concentration after 12 months of therapy was also a predictor of response for male patients. Using these variables, we built a model to predict responsiveness to tafamidis.CONCLUSIONOur study indicates long-term effectiveness for tafamidis, a kinetic stabilizer approved for the treatment of hereditary TTR amyloidosis. Moreover, we created a predictive model that can be potentially used in the clinical setting to inform patients and clinicians in their therapeutic decisions.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationMonteiro C, Mesgazardeh JS, Anselmo J, et al. Predictive model of response to tafamidis in hereditary ATTR polyneuropathy. JCI Insight. 2019;4(12):e126526. Published 2019 Jun 20. doi:10.1172/jci.insight.126526pt_PT
dc.identifier.doi10.1172/jci.insight.126526pt_PT
dc.identifier.issn2379-3708
dc.identifier.urihttp://hdl.handle.net/10400.16/2403
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherAmerican Society for Clinical Investigationpt_PT
dc.relation.publisherversionhttps://insight.jci.org/articles/view/126526pt_PT
dc.subjectGenetic diseasespt_PT
dc.subjectNeuromuscular diseasept_PT
dc.subjectNeurosciencept_PT
dc.subjectProtein misfoldingpt_PT
dc.subjectTherapeuticspt_PT
dc.titlePredictive model of response to tafamidis in hereditary ATTR polyneuropathypt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.conferencePlaceUnited States of Americapt_PT
oaire.citation.issue12pt_PT
oaire.citation.startPagee126526pt_PT
oaire.citation.titleJCI insightpt_PT
oaire.citation.volume4pt_PT
rcaap.rightsopenAccesspt_PT
rcaap.typearticlept_PT

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