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Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis

2020Journal article Open access
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dc.contributor.authorDyck, P. James B.
dc.contributor.authorCoelho, Teresa
dc.contributor.authorWaddington Cruz, Marcia
dc.contributor.authorBrannagan, Thomas H.
dc.contributor.authorKhella, Sami
dc.contributor.authorKaram, Chafic
dc.contributor.authorBerk, John L.
dc.contributor.authorPolydefkis, Michael J.
dc.contributor.authorKincaid, John C.
dc.contributor.authorWiesman, Janice F.
dc.contributor.authorLitchy, William J.
dc.contributor.authorMauermann, Michelle L.
dc.contributor.authorAckermann, Elizabeth J.
dc.contributor.authorBaker, Brenda F.
dc.contributor.authorJung, Shiangtung W.
dc.contributor.authorGuthrie, Spencer
dc.contributor.authorPollock, Michael
dc.contributor.authorDyck, Peter J.
dc.date.accessioned2022-06-30T09:20:41Z
dc.date.available2022-06-30T09:20:41Z
dc.date.issued2020
dc.description.abstractIntroduction: Hereditary transthyretin-mediated amyloidosis (hATTR) manifests as multisystem dysfunction, including progressive polyneuropathy. Inotersen, an antisense oligonucleotide, improved the course of neuropathic impairment in patients with hATTR in the pivotal NEURO-TTR study (NCT01737398). To determine inotersen's impact on symptoms and patients' neuropathy experience, we performed a post hoc analysis of the Neuropathy Symptoms and Change (NSC) score. Methods: Stage 1 or 2 hATTR patients were randomized to receive weekly subcutaneous inotersen or placebo for 65 weeks. NSC score was assessed at baseline and 35 and 66 weeks. Results: At 66 weeks, inotersen-treated patients had symptom stabilization as compared with worsening in patients receiving placebo, based on total NSC score. There were also improvements in the subdomains of muscle weakness, sensory, pain, and autonomic symptoms, and for various individual items. Discussion: Inotersen treatment stabilized neuropathy symptoms, including autonomic symptoms, in patients with hATTR according to NSC score. Thus, the NSC may be an effective measure to assess neuropathy progression and patients' neuropathy experience in clinical practice.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationDyck PJB, Coelho T, Waddington Cruz M, et al. Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis. Muscle Nerve. 2020;62(4):509-515. doi:10.1002/mus.27023pt_PT
dc.identifier.doi10.1002/mus.27023pt_PT
dc.identifier.issn1097-4598
dc.identifier.issn0148-639X
dc.identifier.urihttp://hdl.handle.net/10400.16/2682
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherJohn Wiley & Sonspt_PT
dc.relation.publisherversionhttps://onlinelibrary.wiley.com/doi/10.1002/mus.27023pt_PT
dc.rights.urihttp://creativecommons.org/licenses/by-nc/4.0/pt_PT
dc.subjectNeuropathy Symptoms and Changept_PT
dc.subjectamyloidosispt_PT
dc.subjecthATTRpt_PT
dc.subjectinotersenpt_PT
dc.subjecttransthyretinpt_PT
dc.titleNeuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosispt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.conferencePlaceUnited States of Americapt_PT
oaire.citation.endPage515pt_PT
oaire.citation.issue4pt_PT
oaire.citation.startPage509pt_PT
oaire.citation.titleMuscle & Nervept_PT
oaire.citation.volume62pt_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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