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Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy

2013Journal article Open access
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dc.contributor.authorCoelho, T.
dc.contributor.authorMaia, L.
dc.contributor.authorMartins-Silva, A.
dc.contributor.authorCruz, M.
dc.contributor.authorPlanté-Bordeneuve, V.
dc.contributor.authorSuhr, O.
dc.contributor.authorConceição, I.
dc.contributor.authorSchmidt, H.
dc.contributor.authorTrigo, P.
dc.contributor.authorKelly, J.
dc.contributor.authorLabaudinière, R.
dc.contributor.authorChan, J.
dc.contributor.authorPackman, J.
dc.contributor.authorGrogan, D.
dc.date.accessioned2014-09-22T14:37:24Z
dc.date.available2014-09-22T14:37:24Z
dc.date.issued2013
dc.description.abstractTafamidis, a transthyretin (TTR) kinetic stabilizer, delayed neuropathic progression in patients with Val30Met TTR familial amyloid polyneuropathy (TTR-FAP) in an 18-month randomized controlled trial (study Fx-005). This 12-month, open-label extension study evaluated the long-term safety, tolerability, and efficacy of tafamidis 20 mg once daily in 86 patients who earlier received blinded treatment with tafamidis or placebo. Efficacy measures included the Neuropathy Impairment Score in the Lower Limbs (NIS-LL), Norfolk Quality of Life-Diabetic Neuropathy total quality of life (TQOL) score, and changes in neurologic function and nutritional status. We quantified the monthly rates of change in efficacy measures, and TTR stabilization, and monitored adverse events (AEs). Patients who continued on tafamidis had stable rates of change in NIS-LL (from 0.08 to 0.11/month; p = 0.60) and TQOL (from -0.03 to 0.25; p = 0.16). In patients switched from placebo, the monthly rate of change in NIS-LL declined (from 0.34 to 0.16/month; p = 0.01), as did TQOL score (from 0.61 to -0.16; p < 0.001). Patients treated with tafamidis for 30 months had 55.9 % greater preservation of neurologic function as measured by the NIS-LL than patients in whom tafamidis was initiated later. Plasma TTR was stabilized in 94.1 % of patients treated with tafamidis for 30 months. AEs were similar between groups; no patients discontinued because of an AE. Long-term tafamidis was well tolerated, with the reduced rate of neurologic deterioration sustained over 30 months. Tafamidis also slowed neurologic impairment in patients previously given placebo, but treatment benefits were greater when tafamidis was begun earlier.por
dc.identifier.citationJ Neurol (2013) 260:2802–2814por
dc.identifier.doi10.1007/s00415-013-7051-7
dc.identifier.urihttp://hdl.handle.net/10400.16/1686
dc.language.isoengpor
dc.peerreviewedyespor
dc.publisherSpringer-Verlagpor
dc.relation.publisherversionhttp://link.springer.com/article/10.1007%2Fs00415-013-7051-7por
dc.titleLong-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathypor
dc.typejournal article
dspace.entity.typePublication
oaire.citation.conferencePlaceGermanypor
oaire.citation.endPage2814por
oaire.citation.startPage2802por
oaire.citation.titleJournal of neurologypor
oaire.citation.volume260por
rcaap.rightsopenAccesspor
rcaap.typearticlepor

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