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Tafamidis for trans...
Tafamidis for transthyretin familial amyloid polyneuropathy : A randomized, controlled trial
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Coelho, Teresa (author)
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Maia, Luis F. (author)
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da Silva, Ana Martins (author)
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Cruz, Marcia Waddington (author)
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Plante-Bordeneuve, Violaine (author)
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Lozeron, Pierre (author)
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- Suhr, Ole B. (author)
- Umeå universitet,Medicin
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Campistol, Josep M. (author)
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Conceicao, Isabel Maria (author)
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Schmidt, Hartmut H. -J. (author)
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Trigo, Pedro (author)
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Kelly, Jeffery W. (author)
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Labaudinie, Richard (author)
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Chan, Jason (author)
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Packman, Jeff (author)
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Wilson, Amy (author)
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Grogan, Donna R. (author)
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(creator_code:org_t)
- Philadelphia : Lippincott Williams & Wilkins, 2012
- 2012
- English.
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In: Neurology. - Philadelphia : Lippincott Williams & Wilkins. - 0028-3878 .- 1526-632X. ; 79:8, s. 785-792
- Related links:
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https://urn.kb.se/re...
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https://doi.org/10.1...
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Abstract
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- Objectives: To evaluate the efficacy and safety of 18 months of tafamidis treatment in patients with early-stage V30M transthyretin familial amyloid polyneuropathy (TTR-FAP). Methods: In this randomized, double-blind trial, patients received tafamidis 20 mg QD or placebo. Coprimary endpoints were the Neuropathy Impairment Score-Lower Limbs (NIS-LL) responder analysis (<2-point worsening) and treatment-group difference in the mean change from baseline in Norfolk Quality of Life-Diabetic Neuropathy total score (TQOL) in the intent-to-treat (ITT) population (n = 125). These endpoints were also evaluated in the efficacy-evaluable (EE; n = 87) population. Secondary endpoints, including changes in neurologic function, nutritional status, and TTR stabilization, were analyzed in the ITT population. Results: There was a higher-than-anticipated liver transplantation dropout rate. No differences were observed between the tafamidis and placebo groups for the coprimary endpoints, NIS-LL responder analysis (45.3% vs 29.5% responders; p = 0.068) and change in TQOL (2.0 vs 7.2; p = 0.116) in the ITT population. In the EE population, significantly more tafamidis patients than placebo patients were NIS-LL responders (60.0% vs 38.1%; p = 0.041), and tafamidis patients had better-preserved TQOL (0.1 vs 8.9; p = 0.045). Significant differences in most secondary endpoints favored tafamidis. TTR was stabilized in 98% of tafamidis and 0% of placebo patients (p < 0.0001). Adverse events were similar between groups. Conclusions: Although the coprimary endpoints were not met in the ITT population, tafamidis was associated with no trend toward more NIS-LL responders and a significant reduction in worsening of most neurologic variables, supporting the hypothesis that preventing TTR dissociation can delay peripheral neurologic impairment. Classification of evidence: This study provides Class II evidence that 20 mg tafamidis QD was associated with no difference in clinical progression in patients with TTR-FAP, as measured by the NIS-LL and the Norfolk QOL-DN score. Secondary outcomes demonstrated a significant delay in peripheral neurologic impairment with tafamidis, which was well tolerated over 18 months. Neurology (R) 2012;79:785-792
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Neurologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Neurology (hsv//eng)
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Neurology
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- By the author/editor
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Coelho, Teresa
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Maia, Luis F.
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da Silva, Ana Ma ...
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Cruz, Marcia Wad ...
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Plante-Bordeneuv ...
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Lozeron, Pierre
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show more...
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Suhr, Ole B.
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Campistol, Josep ...
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Conceicao, Isabe ...
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Schmidt, Hartmut ...
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Trigo, Pedro
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Kelly, Jeffery W ...
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Labaudinie, Rich ...
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Chan, Jason
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Packman, Jeff
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Wilson, Amy
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Grogan, Donna R.
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show less...
- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Neurology
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Neurology
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Umeå University