{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,16]],"date-time":"2026-09-16T17:27:50Z","timestamp":1789579670184,"version":"build-2803163510"},"reference-count":40,"publisher":"Wiley","issue":"9","license":[{"start":{"date-parts":[[2024,7,10]],"date-time":"2024-07-10T00:00:00Z","timestamp":1720569600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100006400","name":"Alnylam Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006400","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Euro J of Neurology"],"published-print":{"date-parts":[[2024,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background and purpose<\/jats:title><jats:p>Hereditary transthyretin\u2010mediated amyloidosis with polyneuropathy (ATTRv\u2010PN [v for variant]) is a rare, progressive disease associated with multisystemic impairments. This study assessed the real\u2010world outcomes of patients with ATTRv\u2010PN who switched from tafamidis to patisiran, as well as the reasons for the treatment switch.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This was a retrospective chart review study at a large expert referral center. Data were extracted from medical charts of patients with ATTRv\u2010PN who switched from tafamidis to patisiran on or before 30 August 2019. Data elements included demographic and clinical characteristics, rationale for switch, and disease measures evaluated from tafamidis initiation through the 12\u2010month patisiran treatment period.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Among the 24 patients with ATTRv\u2010PN included in the study, 50.0% had a V30M variant, and the mean (SD) age was 67.3 (8.0) years. During tafamidis treatment (mean [SD] =\u200930.1 [17.5] months) before switching to patisiran, patients worsened across multiple polyneuropathy measures, including walking ability, Neuropathy Impairment Score, and autonomic function. Neuropathic disease progression on tafamidis was the principal reason for switching to patisiran. After 12\u2009months on patisiran (mean [SD] =\u200911.7 [1.4] months), patients experienced attenuated disease progression or improvement in the aforementioned measures of polyneuropathy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Switching from tafamidis to patisiran attenuated the rate of functional decline, and most patients experienced stabilization or improvement of at least one polyneuropathy measure within 12\u2009months of patisiran treatment. Timely switch from tafamidis to patisiran can be beneficial to avoid rapid disease progression in patients with ATTRv\u2010PN.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ene.16384","type":"journal-article","created":{"date-parts":[[2024,7,11]],"date-time":"2024-07-11T06:03:41Z","timestamp":1720677821000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Effectiveness of patisiran after switching from tafamidis for the treatment of hereditary transthyretin\u2010mediated amyloidosis with polyneuropathy"],"prefix":"10.1111","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9765-709X","authenticated-orcid":false,"given":"Celine","family":"Labeyrie","sequence":"first","affiliation":[{"name":"Neurology Department Assistance Publique\u2010Hopitaux de Paris (AP\u2010HP), CERAMIC, Centre Hospitalier Universitaire (CHU) Bic\u00eatre, INSERM U1195, Universit\u00e9 Paris\u2010Saclay  Le Kremlin\u2010Bic\u00eatre 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