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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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- Coelho, Teresa (author)
- Centro Hospitalar Universitário do Porto, Porto, Portugal
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- Waddington Cruz, Márcia (author)
- Hospital Universitário Clementino Fraga Filho, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil
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- Chao, Chi-Chao (author)
- National Taiwan University Hospital, Taipei, Taiwan
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- Parman, Yeşim (author)
- İstanbul Üniversitesi—Istanbul Tıp Fakültesi, Istanbul, Turkey
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- Wixner, Jonas (author)
- Umeå universitet,Avdelningen för medicin
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- Weiler, Markus (author)
- Amyloidosis Center and Department of Neurology, Heidelberg University Hospital, Heidelberg, Germany
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- Barroso, Fabio A. (author)
- Neurology Department, Fleni, Buenos Aires, Argentina
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- Dasgupta, Noel R. (author)
- Indiana University School of Medicine, IN, Indianapolis, United States
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- Jung, Shiangtung W. (author)
- Ionis Pharmaceuticals, Inc., CA, Carlsbad, United States
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- Schneider, Eugene (author)
- Ionis Pharmaceuticals, Inc., CA, Carlsbad, United States
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- Viney, Nicholas J. (author)
- Ionis Pharmaceuticals, Inc., CA, Carlsbad, United States
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- Dyck, P. James B. (author)
- Mayo Clinic, MN, Rochester, United States
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- Ando, Yukio (author)
- Kumamoto University, Kumamoto, Japan
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- Gillmore, Julian D. (author)
- Centre for Amyloidosis, University College London, London, United Kingdom
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- Khella, Sami (author)
- Department of Neurology, University of Pennsylvania School of Medicine, PA, Philadelphia, United States
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- Gertz, Morie A. (author)
- Division of Hematology, Mayo Clinic, MN, Rochester, United States
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- Obici, Laura (author)
- Amyloidosis Research and Treatment Centre, IRCCS Fondazione Policlinico San Matteo, Pavia, Italy
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- Berk, John L. (author)
- Amyloidosis Center, School of Medicine/Boston Medical Center, Boston University, 72 East Concord St., K503, MA, Boston, United States
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(creator_code:org_t)
- 2022-12-16
- 2023
- English.
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In: Neurology and Therapy. - : Springer Nature. - 2193-8253 .- 2193-6536. ; 12, s. 267-287
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Abstract
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- 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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Neurologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Neurology (hsv//eng)
Keyword
- Amyloid
- ATTR
- Cardiomyopathy
- Eplontersen
- Polyneuropathy
- Transthyretin amyloidosis
Publication and Content Type
- ref (subject category)
- art (subject category)
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- By the author/editor
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Coelho, Teresa
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Waddington Cruz, ...
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Chao, Chi-Chao
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Parman, Yeşim
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Wixner, Jonas
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Weiler, Markus
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Barroso, Fabio A ...
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Dasgupta, Noel R ...
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Jung, Shiangtung ...
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Schneider, Eugen ...
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Viney, Nicholas ...
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Dyck, P. James B ...
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Ando, Yukio
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Gillmore, Julian ...
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Khella, Sami
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Gertz, Morie A.
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Obici, Laura
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Berk, John L.
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- MEDICAL AND HEALTH SCIENCES
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and Neurology
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Neurology and Th ...
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Umeå University