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A subset of patients were randomized to receive subcutaneous inotersen 300 mg weekly (Weeks 1\u201334) and subsequently switched to subcutaneous eplontersen 45 mg every 4 weeks (Weeks 37\u201381). Change in serum TTR and treatment-emergent adverse events (TEAEs) were evaluated through Week 85. Effects on neuropathy impairment, QoL, and nutritional status were also evaluated.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Of 24 patients randomized to inotersen, 20 (83%) switched to eplontersen at Week 37 and four discontinued due to AEs\/investigator decision. Absolute change in serum TTR was greater after switching from inotersen (\u221274.3%; Week 35) to eplontersen (\u221280.6%; Week 85). From the end of inotersen treatment, neuropathy impairment and QoL were stable (i.e., did not progress) while on eplontersen, and there was no deterioration in nutritional status. TEAEs were fewer with eplontersen (Weeks 37\u201385; 19\/20 [95%] patients) compared with inotersen (up to Week 35; 24\/24 [100%] patients). Mean platelet counts decreased during inotersen treatment (mean nadir reduction \u201240.7%) and returned to baseline during eplontersen treatment (mean nadir reduction, \u20123.2%).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Switching from inotersen to eplontersen further reduced serum TTR, halted disease progression, stabilized QoL, restored platelet count, and improved tolerability, without deterioration in nutritional status. This supports a positive benefit-risk profile for patients with ATTRv-PN who switch from inotersen to eplontersen.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1007\/s00415-024-12616-6","type":"journal-article","created":{"date-parts":[[2024,8,13]],"date-time":"2024-08-13T14:02:24Z","timestamp":1723557744000},"page":"6655-6666","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":15,"title":["Switching from inotersen to eplontersen in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: analysis from NEURO-TTRansform"],"prefix":"10.1007","volume":"271","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0934-9631","authenticated-orcid":false,"given":"Isabel","family":"Concei\u00e7\u00e3o","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John L.","family":"Berk","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Markus","family":"Weiler","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pedro A.","family":"Kowacs","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Noel R.","family":"Dasgupta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sami","family":"Khella","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chi-Chao","family":"Chao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shahram","family":"Attarian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"T. 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J.L.B. has participated in an Ionis Pharmaceuticals Inc., ad hoc advisory committee. M.W. is a consultant to Akcea Therapeutics, Alnylam Pharmaceuticals, Biogen, Hoffmann-La Roche, Novo Nordisk, Pfizer, and Sobi; and has received speaker fees from Akcea Therapeutics, Alnylam Pharmaceuticals, and Biogen, and financial support for conference attendance from Akcea Therapeutics, Alnylam Pharmaceuticals, Ionis Pharmaceuticals Inc., and Pfizer Inc. P.A.K. is currently participating in the ION trial. N.R.D. is a consultant to Alnylam, Eidos, and Intellia; and has lectured for Alnylam. S.K. is a consultant to Alnylum and Ionis Pharmaceuticals Inc. C-C.C. reports no conflicts of interest. S.A. has provided consultancy for Alnylam Pharmaceuticals, AstraZeneca, and Pfizer Inc. T.J.K., S.W.J., N.J.V., and R.Z.Y. are employees of, and shareholders in Ionis Pharmaceuticals Inc. J.C. is an employee of, and shareholder in AstraZeneca. M.G. has been a consultant to Ionis Pharmaceuticals Inc.\/Akcea, Prothena, Sanofi, Janssen, Aptitude Health, Juno, Physicians Education Resource, AbbVie, Johnson & Johnson, Celgene, Research to Practice, and Sorrento; has received research funding from Ashfield; and has received fees for development of educational materials from i3 Health. A.M. has received research grants from Attralus, Cytokinetics, Ionis Pharmaceuticals Inc., Pfizer, Ultromics, and the Wheeler Foundation; and personal funding from Alnylam, Attralus, Bristol-Myers Squibb, Cytokinetics, Eidos, Haya, Ionis Pharmaceuticals Inc., Pfizer Inc., and Tenaya. M.W.C. is a principal investigator for the NEURO-TTRansform trial and is a consultant for Ionis Pharmaceuticals Inc. T.C. is a principal investigator for the NEURO-TTRansform trial (paid to institution) and has received financial support from Ionis Pharmaceuticals Inc., and Akcea to attend scientific meetings.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}},{"value":"The study protocol was approved by the institutional review board at each participating center, or by an independent ethics committee.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}}]}}