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dc.contributor.authorGertz, Morie A.
dc.contributor.authorParman, Yesim
dc.contributor.authorKhella, Sami
dc.contributor.authorObici, Laura
dc.contributor.authorBerk, John L.
dc.contributor.authorCoelho, Teresa
dc.contributor.authorCruz, Marcia Waddington
dc.contributor.authorChao, Chi-Chao
dc.contributor.authorWixner, Jonas
dc.contributor.authorWeiler, Markus
dc.contributor.authorBarroso, Fabio A.
dc.contributor.authorDasgupta, Noel R.
dc.contributor.authorJung, Shiangtung W.
dc.contributor.authorSchneider, Eugene
dc.contributor.authorViney, Nicholas J.
dc.contributor.authorDyck, P. James B.
dc.contributor.authorAndo, Yukio
dc.contributor.authorGillmore, Julian D.
dc.date.accessioned2023-10-10T10:21:30Z
dc.date.available2023-10-10T10:21:30Z
dc.date.issued2023
dc.identifier.citationCoelho T., Cruz M. W., Chao C., Parman Y., Wixner J., Weiler M., Barroso F. A., Dasgupta N. R., Jung S. W., Schneider E., et al., "Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen", NEUROLOGY AND THERAPY, cilt.12, sa.1, ss.267-287, 2023
dc.identifier.issn2193-8253
dc.identifier.othervv_1032021
dc.identifier.otherav_02ad62b2-77df-4293-8862-e8c6bf5b753d
dc.identifier.urihttp://hdl.handle.net/20.500.12627/189195
dc.identifier.urihttps://doi.org/10.1007/s40120-022-00414-z
dc.identifier.urihttps://avesis.istanbul.edu.tr/api/publication/02ad62b2-77df-4293-8862-e8c6bf5b753d/file
dc.description.abstractIntroduction: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.
dc.language.isoeng
dc.subjectKLİNİK NÖROLOJİ
dc.subjectSağlık Bilimleri
dc.subjectNöroloji
dc.subjectDahili Tıp Bilimleri
dc.subjectTıp
dc.subjectKlinik Tıp (MED)
dc.subjectKlinik Tıp
dc.subjectNöroloji (klinik)
dc.subjectYaşam Bilimleri
dc.titleCharacteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen
dc.typeMakale
dc.relation.journalNEUROLOGY AND THERAPY
dc.contributor.departmentCtr Hosp Univ Porto , ,
dc.identifier.volume12
dc.identifier.issue1
dc.identifier.startpage267
dc.identifier.endpage287
dc.contributor.firstauthorID4309856


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