Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis

Detalhes bibliográficos
Autor(a) principal: Dyck, P. James B.
Data de Publicação: 2020
Outros Autores: Coelho, Teresa, Waddington Cruz, Marcia, Brannagan, Thomas H., Khella, Sami, Karam, Chafic, Berk, John L., Polydefkis, Michael J., Kincaid, John C., Wiesman, Janice F., Litchy, William J., Mauermann, Michelle L., Ackermann, Elizabeth J., Baker, Brenda F., Jung, Shiangtung W., Guthrie, Spencer, Pollock, Michael, Dyck, Peter J.
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Texto Completo: http://hdl.handle.net/10400.16/2682
Resumo: Introduction: 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.
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spelling Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosisNeuropathy Symptoms and ChangeamyloidosishATTRinotersentransthyretinIntroduction: 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.John Wiley & SonsRepositório Científico do Centro Hospitalar Universitário de Santo AntónioDyck, P. James B.Coelho, TeresaWaddington Cruz, MarciaBrannagan, Thomas H.Khella, SamiKaram, ChaficBerk, John L.Polydefkis, Michael J.Kincaid, John C.Wiesman, Janice F.Litchy, William J.Mauermann, Michelle L.Ackermann, Elizabeth J.Baker, Brenda F.Jung, Shiangtung W.Guthrie, SpencerPollock, MichaelDyck, Peter J.2022-06-30T09:20:41Z20202020-01-01T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10400.16/2682engDyck 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.270231097-459810.1002/mus.27023info:eu-repo/semantics/openAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2023-10-20T11:01:40Zoai:repositorio.chporto.pt:10400.16/2682Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-19T20:38:52.666958Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãofalse
dc.title.none.fl_str_mv Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis
title Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis
spellingShingle Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis
Dyck, P. James B.
Neuropathy Symptoms and Change
amyloidosis
hATTR
inotersen
transthyretin
title_short Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis
title_full Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis
title_fullStr Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis
title_full_unstemmed Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis
title_sort Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis
author Dyck, P. James B.
author_facet Dyck, P. James B.
Coelho, Teresa
Waddington Cruz, Marcia
Brannagan, Thomas H.
Khella, Sami
Karam, Chafic
Berk, John L.
Polydefkis, Michael J.
Kincaid, John C.
Wiesman, Janice F.
Litchy, William J.
Mauermann, Michelle L.
Ackermann, Elizabeth J.
Baker, Brenda F.
Jung, Shiangtung W.
Guthrie, Spencer
Pollock, Michael
Dyck, Peter J.
author_role author
author2 Coelho, Teresa
Waddington Cruz, Marcia
Brannagan, Thomas H.
Khella, Sami
Karam, Chafic
Berk, John L.
Polydefkis, Michael J.
Kincaid, John C.
Wiesman, Janice F.
Litchy, William J.
Mauermann, Michelle L.
Ackermann, Elizabeth J.
Baker, Brenda F.
Jung, Shiangtung W.
Guthrie, Spencer
Pollock, Michael
Dyck, Peter J.
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Repositório Científico do Centro Hospitalar Universitário de Santo António
dc.contributor.author.fl_str_mv Dyck, P. James B.
Coelho, Teresa
Waddington Cruz, Marcia
Brannagan, Thomas H.
Khella, Sami
Karam, Chafic
Berk, John L.
Polydefkis, Michael J.
Kincaid, John C.
Wiesman, Janice F.
Litchy, William J.
Mauermann, Michelle L.
Ackermann, Elizabeth J.
Baker, Brenda F.
Jung, Shiangtung W.
Guthrie, Spencer
Pollock, Michael
Dyck, Peter J.
dc.subject.por.fl_str_mv Neuropathy Symptoms and Change
amyloidosis
hATTR
inotersen
transthyretin
topic Neuropathy Symptoms and Change
amyloidosis
hATTR
inotersen
transthyretin
description Introduction: 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.
publishDate 2020
dc.date.none.fl_str_mv 2020
2020-01-01T00:00:00Z
2022-06-30T09:20:41Z
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://hdl.handle.net/10400.16/2682
url http://hdl.handle.net/10400.16/2682
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv Dyck 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.27023
1097-4598
10.1002/mus.27023
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv John Wiley & Sons
publisher.none.fl_str_mv John Wiley & Sons
dc.source.none.fl_str_mv reponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
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