{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T15:13:23Z","timestamp":1793373203753,"version":"4.5.0"},"reference-count":35,"publisher":"American Medical Association (AMA)","issue":"15","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JAMA"],"published-print":{"date-parts":[[2023,10,17]]},"abstract":"\n Importance<\/jats:title>\n Transthyretin gene silencing is an emerging treatment strategy for hereditary transthyretin (ATTRv) amyloidosis.<\/jats:p>\n <\/jats:sec>\n \n Objective<\/jats:title>\n To evaluate eplontersen, an investigational ligand-conjugated antisense oligonucleotide, in ATTRv polyneuropathy.<\/jats:p>\n <\/jats:sec>\n \n Design, Setting, and Participants<\/jats:title>\n \n NEURO-TTRansform was an open-label, single-group, phase 3 trial conducted at 40 sites across 15 countries (December 2019-April 2023) in 168 adults with Coutinho stage 1 or 2 ATTRv polyneuropathy, Neuropathy Impairment Score 10-130, and a documented\n TTR<\/jats:italic>\n variant. Patients treated with placebo from NEURO-TTR (\n NCT01737398<\/jats:ext-link>\n ; March 2013\u2013November 2017), an inotersen trial with similar eligibility criteria and end points, served as a historical placebo (\u201cplacebo\u201d) group.\n <\/jats:p>\n <\/jats:sec>\n \n Interventions<\/jats:title>\n Subcutaneous eplontersen (45 mg every 4 weeks; n\u2009=\u2009144); a small reference group received subcutaneous inotersen (300 mg weekly; n\u2009=\u200924); subcutaneous placebo weekly (in NEURO-TTR; n\u2009=\u200960).<\/jats:p>\n <\/jats:sec>\n \n Main Outcomes and Measures<\/jats:title>\n Primary efficacy end points at week 65\/66 were changes from baseline in serum transthyretin concentration, modified Neuropathy Impairment Score +7 (mNIS+7) composite score (scoring range, \u201322.3 to 346.3; higher scores indicate poorer function), and Norfolk Quality of Life Questionnaire\u2013Diabetic Neuropathy (Norfolk QoL-DN) total score (scoring range, \u20134 to 136; higher scores indicate poorer quality of life). Analyses of efficacy end points were based on a mixed-effects model with repeated measures adjusted by propensity score weights.<\/jats:p>\n <\/jats:sec>\n \n Results<\/jats:title>\n \n Among 144 eplontersen-treated patients (mean age, 53.0 years; 69% male), 136 (94.4%) completed week-66 follow-up; among 60 placebo patients (mean age, 59.5 years; 68% male), 52 (86.7%) completed week-66 follow-up. At week 65, adjusted mean percentage reduction in serum transthyretin was \u221281.7% with eplontersen and \u221211.2% with placebo (difference, \u221270.4% [95% CI, \u221275.2% to \u221265.7%];\n P<\/jats:italic>\n \u2009&amp;lt;\u2009.001). Adjusted mean change from baseline to week 66 was lower (better) with eplontersen vs placebo for mNIS+7 composite score (0.3 vs 25.1; difference, \u221224.8 [95% CI, \u221231.0 to \u221218.6;\n P<\/jats:italic>\n \u2009&amp;lt;\u2009.001) and for Norfolk QoL-DN (\u22125.5 vs 14.2; difference, \u221219.7 [95% CI, \u221225.6 to \u221213.8];\n P<\/jats:italic>\n \u2009&amp;lt;\u2009.001). Adverse events by week 66 that led to study drug discontinuation occurred in 6 patients (4%) in the eplontersen group vs 2 (3%) in the placebo group. Through week 66, there were 2 deaths in the eplontersen group consistent with known disease-related sequelae (cardiac arrhythmia; intracerebral hemorrhage); there were no deaths in the placebo group.\n <\/jats:p>\n <\/jats:sec>\n \n Conclusions and Relevance<\/jats:title>\n In patients with ATTRv polyneuropathy, the eplontersen treatment group demonstrated changes consistent with significantly lowered serum transthyretin concentration, less neuropathy impairment, and better quality of life compared with a historical placebo.<\/jats:p>\n <\/jats:sec>\n \n Trial Registration<\/jats:title>\n \n ClinicalTrials.gov Identifier:\n NCT04136184<\/jats:ext-link>\n ; EU Clinical Trials Register:\n EudraCT 2019-001698-10<\/jats:ext-link>\n <\/jats:p>\n <\/jats:sec>","DOI":"10.1001\/jama.2023.18688","type":"journal-article","created":{"date-parts":[[2023,9,28]],"date-time":"2023-09-28T11:32:56Z","timestamp":1695900776000},"page":"1448","source":"Crossref","is-referenced-by-count":253,"title":["Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy"],"prefix":"10.1001","volume":"330","author":[{"given":"Teresa","family":"Coelho","sequence":"first","affiliation":[{"name":"Centro Hospitalar Universit\u00e1rio de Santo Ant\u00f3nio, Porto, Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"suffix":"Jr","given":"Wilson","family":"Marques","sequence":"additional","affiliation":[{"name":"Hospital das Cl\u00ednicas da Faculdade de Medicina de Ribeir\u00e3o Preto, Ribeir\u00e3o Preto, Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Noel R.","family":"Dasgupta","sequence":"additional","affiliation":[{"name":"Indiana University School of Medicine, 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