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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

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dc.contributor.author Brannagan, Thomas H.
dc.contributor.author Wang, Annabel K.
dc.contributor.author Coelho, Teresa
dc.contributor.author Waddington Cruz, Marcia
dc.contributor.author Polydefkis, Michael J.
dc.contributor.author Dyck, Peter J.
dc.contributor.author Plante-Bordeneuve, Violaine
dc.contributor.author Berk, John L.
dc.contributor.author Barroso, Fabio Adrián
dc.contributor.author Merlini, Giampaolo
dc.contributor.author Conceição, Isabel
dc.contributor.author Hughes, Steven G.
dc.contributor.author Kwoh, Jesse
dc.contributor.author Jung, Shiangtung W.
dc.contributor.author Guthrie, Spencer
dc.contributor.author Pollock, Michael
dc.contributor.author Benson, Merrill D.
dc.contributor.author Gertz, Morie
dc.contributor.author NEURO-TTR open-label extension investigators
dc.date.accessioned 2020-11-12T13:42:30Z
dc.date.available 2020-11-12T13:42:30Z
dc.date.issued 2020-08
dc.identifier.citation Brannagan, T.H., Wang, A.K., Coelho, T., Waddington Cruz, M., Polydefkis, M.J., Dyck, P.J., Plante-Bordeneuve, V., Berk, J.L., Barroso, F., Merlini, G., Conceição, I., Hughes, S.G., Kwoh, J., Jung, S.W., Guthrie, S., Pollock, M., Benson, M.D., Gertz, M., 2020. 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. Eur J Neurol. https://doi.org/10.1111/ene.14285 en_US
dc.identifier.uri https://doi.org/10.1111/ene.14285
dc.identifier.uri https://repositorio.fleni.org.ar/handle/123456789/230
dc.description.abstract Background 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. en_US
dc.language.iso eng en_US
dc.publisher Wiley en_US
dc.rights info:eu-repo/semantics/openAccess
dc.rights.uri https://creativecommons.org/licenses/by/2.5/ar/
dc.subject Amyloidosis en_US
dc.subject Amiloidosis en_US
dc.subject Polyneuropathies en_US
dc.subject Polineuropatías en_US
dc.title 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 en_US
dc.type info:eu-repo/semantics/article en_US
dc.type info:eu-repo/semantics/publishedVersion
dc.description.fil Fil: Barroso, Fabio Adrián. Fleni. Departamento de Neurología. Sección de Enfermedades Neuromusculares; Argentina.
dc.description.fil Fil: Brannagan, Thomas H. Columbia University Medical Center; Estados Unidos.
dc.description.fil Fil: Wang, Annabel K. University of California; Estados Unidos.
dc.description.fil Fil: Coelho, Teresa. Centro Hospitalar do Porto; Portugal.
dc.description.fil Fil: Waddington Cruz, Marcia. Federal University of Rio de Janeiro. Amyloidosis Referral Center. CEPARM; Brasil.
dc.description.fil Fil: Polydefkis, Michael J. Johns Hopkins University; Estados Unidos.
dc.description.fil Fil: Dyck, Peter J. Mayo Clinic; Estados Unidos.
dc.description.fil Fil: Plante-Bordeneuve Violaine. CHU Henri Mondor; Francia.
dc.description.fil Fil: Berk, John L. Boston University; Estados Unidos.
dc.description.fil Fil: Merlini, Giampaolo. University of Pavia. IRCCS Policlinico San Matteo. Amyloidosis Center; Italia.
dc.description.fil Fil: Conceição, Isabel. Universidade de Lisboa. Hospital Santa Maria and Faculdade de Medicina. CHULN; Portugal.
dc.description.fil Fil: Hughes, Steven G. Ionis Pharmaceuticals, Inc; Estados Unidos.
dc.description.fil Fil: Kwoh, Jesse. Ionis Pharmaceuticals, Inc; Estados Unidos.
dc.description.fil Fil: Jung, Shiangtung W. Ionis Pharmaceuticals, Inc.; Estados Unidos.
dc.description.fil Fil: Guthrie, Spencer. Aurora Bio; Estados Unidos.
dc.description.fil Fil: Pollock, Michael. Akcea Therapeutics, Inc.; Estados Unidos.
dc.description.fil Fil: Benson, Merrill D. Indiana University School of Medicine; Estados Unidos.
dc.description.fil Fil: Gertz, Morie. Mayo Clinic; Estados Unidos.
dc.relation.ispartofVOLUME 27
dc.relation.ispartofNUMBER 8
dc.relation.ispartofPAGINATION 1374-1381.
dc.relation.ispartofCOUNTRY Estados Unidos
dc.relation.ispartofCITY Nueva York
dc.relation.ispartofTITLE European journal of neurology
dc.relation.ispartofISSN 1468-1331
dc.type.snrd info:ar-repo/semantics/artículo


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