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Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen

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dc.contributor.author Coelho, Teresa
dc.contributor.author Waddington Cruz, Marcia
dc.contributor.author Chao, Chi-Chao
dc.contributor.author Parman, Yeşim
dc.contributor.author Wixner, Jonas
dc.contributor.author Weiler, Markus
dc.contributor.author Barroso, Fabio Adrián
dc.contributor.author Dasgupta, Noel R.
dc.contributor.author Jung, Shiangtung W.
dc.contributor.author Schneider, Eugene
dc.contributor.author Viney, Nicholas J.
dc.contributor.author Dyck, P. James B.
dc.contributor.author Ando, Yukio
dc.contributor.author Gillmore, Julian D.
dc.contributor.author Khella, Sami
dc.contributor.author Gertz, Morie
dc.contributor.author Obici, Laura
dc.contributor.author Berk, John L.
dc.date.accessioned 2023-01-06T11:37:18Z
dc.date.available 2023-01-06T11:37:18Z
dc.date.issued 2022-12-16
dc.identifier.citation Coelho T, Waddington Cruz M, Chao CC, Parman Y, Wixner J, Weiler M, Barroso FA, Dasgupta NR, Jung SW, Schneider E, Viney NJ, Dyck PJB, Ando Y, Gillmore JD, Khella S, Gertz M, Obici L, Berk JL. Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen. Neurol Ther. 2022 Dec 16. doi: 10.1007/s40120-022-00414-z. Epub ahead of print. es_ES
dc.identifier.uri https://doi.org/10.1007/s40120-022-00414-z
dc.identifier.uri https://repositorio.fleni.org.ar/xmlui/handle/123456789/759
dc.description.abstract Introduction: Hereditary transthyretin (ATTRv) amyloidosis is a rare, severe, progressive, debilitating, and ultimately fatal disease caused by systemic deposition of transthyretin (TTR) amyloid fibrils. ATTRv amyloidosis occurs in both males and females. Eplontersen (ION-682884), a ligand-conjugated antisense oligonucleotide designed to degrade hepatic TTR mRNA, is being evaluated for the treatment of ATTRv amyloidosis with polyneuropathy (ATTRv-PN) in the phase 3, international, multicenter, open-label NEURO-TTRansform study (NCT04136184). To describe the study population of this pivotal trial, here we report the baseline characteristics of patients enrolled in the NEURO-TTRansform study. Methods: Patients eligible for NEURO-TTRansform were 18-82 years old with a diagnosis of ATTRv-PN and Coutinho stage 1 (ambulatory without assistance) or stage 2 (ambulatory with assistance) disease; documented TTR gene variant; signs and symptoms consistent with neuropathy associated with ATTRv; no prior liver transplant; and New York Heart Association (NYHA) functional class I or II. Results: The NEURO-TTRansform study enrolled 168 patients across 15 countries/territories (North America, 15.5%; Europe, 38.1%; South America/Australia/Asia, 46.4%). At baseline, the study cohort had a mean age of 52.8 years, 69.0% of patients were male, and 78.0% of patients were White. The V30M variant was most prevalent (60.1% of patients), and prevalence varied by region. Overall, 56.5% and 17.3% of patients had received previous treatment with tafamidis or diflunisal, respectively. A majority of patients (79.2%) had Coutinho stage 1 disease (unimpaired ambulation) and early (before age 50) disease onset (53.0%). Time from diagnosis to enrollment was 46.6 (57.4) months (mean [standard deviation]). Most patients had a baseline polyneuropathy disability (PND) score of I (40.5%) or II (41.1%), and the mean modified Neuropathy Impairment Score + 7 (mNIS + 7) was 79.0. Conclusion: The recruited population in the ongoing NEURO-TTRansform study has global representation characteristic of contemporary clinical practice. es_ES
dc.language.iso eng es_ES
dc.publisher Springer es_ES
dc.rights info:eu-repo/semantics/openAccess
dc.rights.uri https://creativecommons.org/licenses/by/2.5/ar/
dc.subject Cardiomyopathies es_ES
dc.subject Cardiomiopatías es_ES
dc.subject Amiloidosis es_ES
dc.subject Amyloidosis es_ES
dc.subject Neuropatías Amiloides es_ES
dc.subject Amyloid Neuropathies es_ES
dc.title Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen es_ES
dc.type info:eu-repo/semantics/article es_ES
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: Coelho, Teresa. Centro Hospitalar Universitário do Porto; Portugal.
dc.description.fil Fil: Waddington Cruz, Marcia. Federal University of Rio de Janeiro; Brasil.
dc.description.fil Fil: Chao, Chi-Chao. National Taiwan University Hospital; Taiwan.
dc.description.fil Fil: Parman, Yeşim. İstanbul Üniversitesi-Istanbul Tıp Fakültesi; Turquía.
dc.description.fil Fil: Wixner, Jonas. Umeå University; Suiza.
dc.description.fil Fil: Weiler, Markus. Heidelberg University Hospital; Alemania.
dc.description.fil Fil: Dasgupta, Noel R. Indiana University School of Medicine; Estados Unidos.
dc.description.fil Fil: Jung, Shiangtung W. Ionis Pharmaceuticals, Inc.; Estados Unidos.
dc.description.fil Fil: Schneider, Eugene. Ionis Pharmaceuticals, Inc.; Estados Unidos.
dc.description.fil Fil: Viney, Nicholas J. Ionis Pharmaceuticals, Inc.; Estados Unidos.
dc.description.fil Fil: Dyck, P. James B. Mayo Clinic; Estados Unidos.
dc.description.fil Fil: Ando, Yukio. Kumamoto University; Japón.
dc.description.fil Fil: Gillmore, Julian D. University College London; Reino Unido.
dc.description.fil Fil: Khella, Sami. University of Pennsylvania School of Medicine; Estados Unidos.
dc.description.fil Fil: Gertz, Morie. Mayo Clinic; Estados Unidos.
dc.description.fil Fil: Obici, Laura. IRCCS Fondazione Policlinico San Matteo; Italia.
dc.description.fil Fil: Berk, John L. Boston University. School of Medicine/Boston Medical Center; Estados Unidos.
dc.relation.ispartofCOUNTRY Estados Unidos
dc.relation.ispartofCITY Nueva York
dc.relation.ispartofTITLE Neurology and therapy
dc.relation.ispartofISSN 2193-6536
dc.type.snrd info:ar-repo/semantics/artículo es_ES


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