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Early data on long‐term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2‐year update from the open‐label extension of the NEURO‐TTR trial

2020Journal article Open access
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dc.contributor.authorBrannagan, T. H.
dc.contributor.authorWang, A. K.
dc.contributor.authorCoelho, T.
dc.contributor.authorWaddington Cruz, M.
dc.contributor.authorPolydefkis, M. J.
dc.contributor.authorDyck, P. J.
dc.contributor.authorPlante‐Bordeneuve, V.
dc.contributor.authorBerk, J. L.
dc.contributor.authorBarroso, F.
dc.contributor.authorMerlini, G.
dc.contributor.authorConceição, I.
dc.contributor.authorHughes, S. G.
dc.contributor.authorKwoh, J.
dc.contributor.authorJung, S. W.
dc.contributor.authorGuthrie, S.
dc.contributor.authorPollock, M.
dc.contributor.authorBenson, M. D.
dc.contributor.authorGertz, M.
dc.contributor.authorDrachman, Brian
dc.contributor.authorGorevic, Peter
dc.contributor.authorHeitner, Stephen
dc.contributor.authorScheinberg, Morton
dc.contributor.authorSchmidt, Hartmut
dc.contributor.authorWhelan, Carol
dc.contributor.authorAdams, David
dc.contributor.authorCampistol Plana, Josep Maria
dc.contributor.authorGamez, Josep
dc.contributor.authorGane, Edward
dc.contributor.authorKristen, Arnt
dc.contributor.authorObici, Laura
dc.contributor.authorSalvi, Fabrizio
dc.contributor.authorSouza Bulle Oliveira, Acary
dc.contributor.authorVita, Giuseppe
dc.date.accessioned2021-11-23T11:17:04Z
dc.date.available2021-11-23T11:17:04Z
dc.date.issued2020
dc.description.abstractBackground and purpose: Hereditary transthyretin (hATTR) amyloidosis causes progressive polyneuropathy resulting from transthyretin (TTR) amyloid deposition throughout the body, including the peripheral nerves. The efficacy and safety of inotersen, an antisense oligonucleotide inhibitor of TTR protein production, were demonstrated in the pivotal NEURO-TTR study in patients with hATTR polyneuropathy. Here, the long-term efficacy and safety of inotersen are assessed in an ongoing open-label extension (OLE) study. Methods: Patients who completed NEURO-TTR were eligible to enroll in the OLE (NCT02175004). Efficacy assessments included the modified Neuropathy Impairment Score plus seven neurophysiological tests composite score (mNIS + 7), the Norfolk Quality of Life - Diabetic Neuropathy (Norfolk QOL-DN) questionnaire total score and the Short-Form 36 Health Survey (SF-36) Physical Component Summary (PCS) score. Safety and tolerability were also assessed. Results: Overall, 97% (135/139) of patients who completed NEURO-TTR enrolled in the OLE. Patients who received inotersen for 39 cumulative months in NEURO-TTR and the OLE continued to show benefit; patients who switched from placebo to inotersen in the OLE demonstrated improvement or stabilization of neurological disease progression by mNIS + 7, Norfolk QOL-DN and SF-36 PCS. No new safety concerns were identified. There was no evidence of increased risk for grade 4 thrombocytopenia or severe renal events with increased duration of inotersen exposure. Conclusion: Inotersen slowed disease progression and reduced deterioration of quality of life in patients with hATTR polyneuropathy. Early treatment with inotersen resulted in greater long-term disease stabilization than delayed initiation. Routine platelet and renal safety monitoring were effective; no new safety signals were observed.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationBrannagan TH, Wang AK, Coelho T, et al. Early data on long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2-year update from the open-label extension of the NEURO-TTR trial. Eur J Neurol. 2020;27(8):1374-1381. doi:10.1111/ene.14285pt_PT
dc.identifier.doi10.1111/ene.14285pt_PT
dc.identifier.issn1351-5101
dc.identifier.issn1468-1331
dc.identifier.urihttp://hdl.handle.net/10400.16/2612
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherWiley-Blackwellpt_PT
dc.relation.publisherversionhttps://onlinelibrary.wiley.com/doi/10.1111/ene.14285pt_PT
dc.rights.urihttp://creativecommons.org/licenses/by-nc/4.0/pt_PT
dc.subjectgenetic and inherited disorderspt_PT
dc.subjectperipheral neuropathiespt_PT
dc.subjectpolyneuropathypt_PT
dc.titleEarly data on long‐term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2‐year update from the open‐label extension of the NEURO‐TTR trialpt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.conferencePlaceEnglandpt_PT
oaire.citation.endPage1381pt_PT
oaire.citation.issue8pt_PT
oaire.citation.startPage1374pt_PT
oaire.citation.titleEuropean Journal of Neurologypt_PT
oaire.citation.volume27pt_PT
rcaap.rightsopenAccesspt_PT
rcaap.typearticlept_PT

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