{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,11]],"date-time":"2026-06-11T03:24:54Z","timestamp":1781148294433,"version":"3.54.1"},"reference-count":26,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2013,7,9]],"date-time":"2013-07-09T00:00:00Z","timestamp":1373328000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Euro J of Neurology"],"published-print":{"date-parts":[[2013,12]]},"abstract":"<jats:sec><jats:title>Background and purpose<\/jats:title><jats:p>The aim of this study was to assess the effect of Tafamidis, which slows the progression of early stages of Met30 transthyretin (<jats:styled-content style=\"fixed-case\">TTR<\/jats:styled-content>) familial amyloidosis polyneuropathy (<jats:styled-content style=\"fixed-case\">FAP<\/jats:styled-content>) in more advanced cases.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The study was a prospective, non\u2010randomized controlled trial carried out at the French national reference centre for <jats:styled-content style=\"fixed-case\">FAP<\/jats:styled-content> with follow\u2010up at 1\u00a0year. Thirty\u2010seven consecutive Met30\u2010<jats:styled-content style=\"fixed-case\">TTR<\/jats:styled-content>\u2010<jats:styled-content style=\"fixed-case\">FAP<\/jats:styled-content> patients were enrolled between December 2009 and July 2011, with <jats:styled-content style=\"fixed-case\">NIS<\/jats:styled-content>\u2010<jats:styled-content style=\"fixed-case\">LL<\/jats:styled-content> (Neuropathy Impairment Score\u2010lower limbs) &gt;\u00a010 and Karnofsky score\u00a0&gt;\u00a060. Their mean (SD) age was 56.4\u00a0 (19) years. Seventy\u2010seven per cent of patients had a walking disability. Seven patients (19%) were withdrawn for adverse effects. The primary study outcome measurements, planned before data collection began, were <jats:styled-content style=\"fixed-case\">NIS<\/jats:styled-content>\u2010<jats:styled-content style=\"fixed-case\">LL<\/jats:styled-content> and <jats:styled-content style=\"fixed-case\">NIS<\/jats:styled-content>\u2010<jats:styled-content style=\"fixed-case\">UL<\/jats:styled-content> (upper limbs) scores and disability scores.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Of the 37 patients entered into the study, 29 were evaluated at 6\u00a0months and 13 at 12\u00a0months. During the first 6\u00a0months of treatment, the mean progression of <jats:styled-content style=\"fixed-case\">NIS<\/jats:styled-content>\u2010<jats:styled-content style=\"fixed-case\">LL<\/jats:styled-content> score was 4.8 and was similar to that during the period before treatment. Among the 45% of patients without <jats:styled-content style=\"fixed-case\">NIS<\/jats:styled-content>\u2010<jats:styled-content style=\"fixed-case\">LL<\/jats:styled-content> progression, the <jats:styled-content style=\"fixed-case\">NIS<\/jats:styled-content>\u2010<jats:styled-content style=\"fixed-case\">UL<\/jats:styled-content> score worsened in 55%. During the first year, 55% deteriorated with respect to disability and 38% with respect to <jats:styled-content style=\"fixed-case\">NIS<\/jats:styled-content> only; only two patients (7%) remained stable. Four (out of 20; 20%) patients who were previously stage 1 reached stage 2 (walking with aid) after this period. Two out of nine patients who were initially normotensive developed orthostatic hypotension. There were a total of 19 adverse events, including four febrile urinary tract infections and three severe diarrhoeas, with faecal incontinence in two.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>In most patients with advanced Met30 <jats:styled-content style=\"fixed-case\">TTR<\/jats:styled-content>\u2010<jats:styled-content style=\"fixed-case\">FAP<\/jats:styled-content>, Tafamidis is not able to stop disease progression, in respect of both <jats:styled-content style=\"fixed-case\">NIS<\/jats:styled-content>\u2010<jats:styled-content style=\"fixed-case\">LL<\/jats:styled-content> and disability. Other anti\u2010amyloid medicines should be assessed in this context.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ene.12225","type":"journal-article","created":{"date-parts":[[2013,7,9]],"date-time":"2013-07-09T01:10:46Z","timestamp":1373332246000},"page":"1539-1545","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":115,"title":["Effect on disability and safety of Tafamidis in late onset of Met30 transthyretin familial amyloid polyneuropathy"],"prefix":"10.1111","volume":"20","author":[{"given":"P.","family":"Lozeron","sequence":"first","affiliation":[{"name":"Department of Neurology APHP, CHU de Bic\u00eatre Le Kremlin Bic\u00eatre France"},{"name":"APHP, CHU de Bic\u00eatre, French national reference centre for FAP (NNERF) Le Kremlin Bic\u00eatre France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M.","family":"Th\u00e9audin","sequence":"additional","affiliation":[{"name":"Department of Neurology APHP, CHU de Bic\u00eatre Le Kremlin Bic\u00eatre France"},{"name":"APHP, CHU de Bic\u00eatre, French national reference centre for FAP (NNERF) Le Kremlin Bic\u00eatre France"},{"name":"INSERM U788 APHP, CHU de Bic\u00eatre Le Kremlin Bic\u00eatre France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Z.","family":"Mincheva","sequence":"additional","affiliation":[{"name":"Department of Neurology APHP, CHU de Bic\u00eatre Le Kremlin Bic\u00eatre France"},{"name":"APHP, CHU de Bic\u00eatre, French national reference centre for FAP (NNERF) Le Kremlin Bic\u00eatre France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B.","family":"Ducot","sequence":"additional","affiliation":[{"name":"INSERM Unit\u00e9 822, IFR69; INED APHP, CHU de Bic\u00eatre Le Kremlin Bic\u00eatre France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C.","family":"Lacroix","sequence":"additional","affiliation":[{"name":"APHP, CHU de Bic\u00eatre, French national reference centre for FAP (NNERF) Le Kremlin Bic\u00eatre France"},{"name":"Department of Neuropathology APHP, CHU de Bic\u00eatre Le Kremlin Bic\u00eatre France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"D.","family":"Adams","sequence":"additional","affiliation":[{"name":"Department of Neurology APHP, CHU de Bic\u00eatre Le Kremlin Bic\u00eatre France"},{"name":"APHP, CHU de Bic\u00eatre, French national reference centre for FAP (NNERF) Le Kremlin Bic\u00eatre France"},{"name":"INSERM U788 APHP, CHU de Bic\u00eatre Le Kremlin Bic\u00eatre France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"French Network for FAP (CORNAMYL)","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2013,7,9]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1007\/s004150170109"},{"key":"e_1_2_7_3_1","first-page":"88","volume-title":"Amyloid Ans Amyloidosis","author":"Coutinho P","year":"1980"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/0140-6736(93)93127-M"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/123.7.1495"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1600-6143.2007.01969.x"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e318248df18"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.3109\/13506129.2011.574354072"},{"key":"e_1_2_7_9_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3181ec7fda"},{"key":"e_1_2_7_10_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10072-012-1105-y"},{"key":"e_1_2_7_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1529-8027.2011.00331.x"},{"key":"e_1_2_7_12_1","doi-asserted-by":"publisher","DOI":"10.3109\/13506129.2012.685665"},{"key":"e_1_2_7_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jmb.2011.12.060"},{"key":"e_1_2_7_14_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3182661eb1"},{"key":"e_1_2_7_15_1","doi-asserted-by":"publisher","DOI":"10.1159\/000052074"},{"key":"e_1_2_7_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.0954-6820.1983.tb08613.x"},{"key":"e_1_2_7_17_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-007-0617-5"},{"key":"e_1_2_7_18_1","doi-asserted-by":"publisher","DOI":"10.1002\/1097-0142(19800415)45:8<2220::AID-CNCR2820450835>3.0.CO;2-Q"},{"key":"e_1_2_7_19_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.46.5.1470"},{"key":"e_1_2_7_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.410380323"},{"key":"e_1_2_7_21_1","unstructured":"European Medicines Agency.http:\/\/www.ema.europa.eu\/docs\/en_GB\/document_library\/EPAR_-_Public_assessment_report\/human\/002294\/WC500117838.pdf. 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