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Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis

2020Journal article Open access
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dc.contributor.authorCoelho, Teresa
dc.contributor.authorYarlas, Aaron
dc.contributor.authorWaddington-Cruz, Marcia
dc.contributor.authorWhite, Michelle K.
dc.contributor.authorSikora Kessler, Asia
dc.contributor.authorLovley, Andrew
dc.contributor.authorPollock, Michael
dc.contributor.authorGuthrie, Spencer
dc.contributor.authorAckermann, Elizabeth J.
dc.contributor.authorHughes, Steven G.
dc.contributor.authorKaram, Chafic
dc.contributor.authorKhella, Sami
dc.contributor.authorGertz, Morie
dc.contributor.authorMerlini, Giampaolo
dc.contributor.authorObici, Laura
dc.contributor.authorSchmidt, Hartmut H.
dc.contributor.authorPolydefkis, Michael
dc.contributor.authorDyck, P. James B.
dc.contributor.authorBrannagan III, Thomas H.
dc.contributor.authorConceição, Isabel
dc.contributor.authorBenson, Merrill D.
dc.contributor.authorBerk, John L.
dc.date.accessioned2022-03-21T12:01:05Z
dc.date.available2022-03-21T12:01:05Z
dc.date.issued2020
dc.description.abstractObjective: To examine the impact on quality of life (QOL) of patients with hATTR amyloidosis with polyneuropathy treated with inotersen (Tegsedi™) versus placebo. Methods: Data were from the NEURO-TTR trial (ClinicalTrials.gov Identifier: NCT01737398), a phase 3, multinational, randomized, double-blind, placebo-controlled study of inotersen in patients with hATTR amyloidosis with polyneuropathy. At baseline and week 66, QOL measures-the Norfolk-QOL-Diabetic Neuropathy (DN) questionnaire and SF-36v2® Health Survey (SF-36v2)-were assessed. Treatment differences in mean changes in QOL from baseline to week 66 were tested using mixed-effect models with repeated measures. Responder analyses compared the percentages of patients whose QOL meaningfully improved or worsened from baseline to week 66 in inotersen and placebo arms. Descriptive analysis of item responses examined treatment differences in specific activities and functions at week 66. Results: Statistically significant mean differences between treatment arms were observed for three of five Norfolk-QOL-DN domains and five of eight SF-36v2 domains, with better outcomes for inotersen than placebo in physical functioning, activities of daily living, neuropathic symptoms, pain, role limitations due to health problems, and social functioning. A larger percentage of patients in the inotersen arm than the placebo arm showed preservation or improvement in Norfolk-QOL-DN and SF-36v2 scores from baseline to week 66. Responses at week 66 showed more substantial problems with daily activities and functioning for patients in the placebo arm than in the inotersen arm. Conclusion: Patients with hATTR amyloidosis with polyneuropathy treated with inotersen showed preserved or improved QOL at 66 weeks compared to those who received placebo.pt_PT
dc.description.sponsorshipThis research was funded by Akcea Therapeutics and Ionis Pharmaceuticals, Incpt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationCoelho T, Yarlas A, Waddington-Cruz M, et al. Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis. J Neurol. 2020;267(4):1070-1079. doi:10.1007/s00415-019-09671-9pt_PT
dc.identifier.doi10.1007/s00415-019-09671-9pt_PT
dc.identifier.issn0340-5354
dc.identifier.issn1432-1459
dc.identifier.urihttp://hdl.handle.net/10400.16/2665
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherSpringer-Verlagpt_PT
dc.relation.publisherversionhttps://link.springer.com/article/10.1007/s00415-019-09671-9pt_PT
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/pt_PT
dc.subjectPhysical functionpt_PT
dc.subjectPolyneuropathypt_PT
dc.subjectQuality of lifept_PT
dc.subjectRare diseasept_PT
dc.subjectTransthyretin amyloidosispt_PT
dc.titleInotersen preserves or improves quality of life in hereditary transthyretin amyloidosispt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.conferencePlaceGermanypt_PT
oaire.citation.endPage1079pt_PT
oaire.citation.issue4pt_PT
oaire.citation.startPage1070pt_PT
oaire.citation.titleJournal of Neurologypt_PT
oaire.citation.volume267pt_PT
rcaap.rightsopenAccesspt_PT
rcaap.typearticlept_PT

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