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Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years

2020Journal article Open access
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dc.contributor.authorMerlini, Giampaolo
dc.contributor.authorCoelho, Teresa
dc.contributor.authorWaddington Cruz, Márcia
dc.contributor.authorLi, Huihua
dc.contributor.authorStewart, Michelle
dc.contributor.authorEbede, Ben
dc.date.accessioned2022-06-30T10:50:12Z
dc.date.available2022-06-30T10:50:12Z
dc.date.issued2020
dc.description.abstractIntroduction: The effects of tafamidis on mortality in Val30Met and non-Val30Met patients with transthyretin amyloidosis with polyneuropathy (ATTR-PN) were evaluated. Methods: The analyses were based on cumulative data from the Val30Met patients in the 18-month double-blind registration study and its 12-month open-label extension study, the non-Val30Met patients of the 12-month open-label study, and both patient groups in the ongoing 10-year extension study. Kaplan-Meier analyses of time to death from first treatment dose were performed. For the Val30Met group, two treatment groups were analyzed: those who received tafamidis in both the parent and extension studies (T-T) and those who received placebo in the parent study and switched to tafamidis in the extension studies (P-T). Results: Kaplan-Meier estimates (95% confidence interval [CI]) were available up to 9 years for the Val30Met group, at which time 85.9% (53.1-96.4) and 91.1% (77.9-96.6) of the patients in the T-T and P-T groups, respectively, were alive. For the non-Val30Met group, estimates were available up to 8 years from the first dose, and the percentage of patients alive was 75.9% (47.7-90.2). Conclusion: Long-term tafamidis treatment may confer survival benefit in patients with ATTR-PN.pt_PT
dc.description.sponsorshipThese studies were initially sponsored by FoldRx Pharmaceuticals, which was acquired by Pfizer in October 2010. The current analyses were sponsored by Pfizer. Funding for the journal’s Rapid Service was provided by Pfizer. All authors had full access to all of the data in this study and take complete responsibility for the integrity of the data and accuracy of the data analysis.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationMerlini G, Coelho T, Waddington Cruz M, Li H, Stewart M, Ebede B. Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years. Neurol Ther. 2020;9(1):105-115. doi:10.1007/s40120-020-00180-wpt_PT
dc.identifier.doi10.1007/s40120-020-00180-wpt_PT
dc.identifier.issn2193-8253
dc.identifier.issn2193-6536
dc.identifier.urihttp://hdl.handle.net/10400.16/2685
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherSpringerpt_PT
dc.relation.publisherversionhttps://link.springer.com/article/10.1007/s40120-020-00180-wpt_PT
dc.rights.urihttp://creativecommons.org/licenses/by-nc/4.0/pt_PT
dc.subjectAmyloidosis; Mortalitypt_PT
dc.subjectPolyneuropathypt_PT
dc.subjectTafamidispt_PT
dc.subjectTransthyretinpt_PT
dc.subjectVal30Metpt_PT
dc.titleEvaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Yearspt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.conferencePlaceNew Zealandpt_PT
oaire.citation.endPage115pt_PT
oaire.citation.issue1pt_PT
oaire.citation.startPage105pt_PT
oaire.citation.titleNeurology and Therapypt_PT
oaire.citation.volume9pt_PT
person.familyNameCoelho
person.givenNameTeresa
person.identifier.ciencia-idC71E-3343-F445
rcaap.rightsopenAccesspt_PT
rcaap.typearticlept_PT
relation.isAuthorOfPublicationca69c0a3-d48a-4b92-bbf7-c17288d609b7
relation.isAuthorOfPublication.latestForDiscoveryca69c0a3-d48a-4b92-bbf7-c17288d609b7

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