PMID- 23974642
OWN - NLM
STAT- MEDLINE
DCOM- 20140618
LR  - 20211021
IS  - 1432-1459 (Electronic)
IS  - 0340-5354 (Print)
IS  - 0340-5354 (Linking)
VI  - 260
IP  - 11
DP  - 2013 Nov
TI  - Long-term effects of tafamidis for the treatment of transthyretin familial 
      amyloid polyneuropathy.
PG  - 2802-14
LID - 10.1007/s00415-013-7051-7 [doi]
AB  - Tafamidis, 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.
FAU - Coelho, Teresa
AU  - Coelho T
AD  - Unidade Clinica de Paramiloidose, Hospital de Santo Antonio, Largo Prof Abel 
      Salazar, 4099-001, Porto, Portugal, tcoelho@netcabo.pt.
FAU - Maia, Luis F
AU  - Maia LF
FAU - da Silva, Ana Martins
AU  - da Silva AM
FAU - Cruz, Marcia W
AU  - Cruz MW
FAU - Plante-Bordeneuve, Violaine
AU  - Plante-Bordeneuve V
FAU - Suhr, Ole B
AU  - Suhr OB
FAU - Conceicao, Isabel
AU  - Conceicao I
FAU - Schmidt, Hartmut H-J
AU  - Schmidt HH
FAU - Trigo, Pedro
AU  - Trigo P
FAU - Kelly, Jeffery W
AU  - Kelly JW
FAU - Labaudiniere, Richard
AU  - Labaudiniere R
FAU - Chan, Jason
AU  - Chan J
FAU - Packman, Jeff
AU  - Packman J
FAU - Grogan, Donna R
AU  - Grogan DR
LA  - eng
SI  - ClinicalTrials.gov/NCT00791492
GR  - R37 DK046335/DK/NIDDK NIH HHS/United States
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
DEP - 20130822
PL  - Germany
TA  - J Neurol
JT  - Journal of neurology
JID - 0423161
RN  - 0 (Benzoxazoles)
RN  - 0 (Prealbumin)
RN  - 8FG9H9D31J (tafamidis)
RN  - AE28F7PNPL (Methionine)
RN  - HG18B9YRS7 (Valine)
SB  - IM
MH  - Adult
MH  - Aged
MH  - Amyloid Neuropathies, Familial/*drug therapy/genetics/physiopathology/psychology
MH  - Benzoxazoles/*therapeutic use
MH  - Disease Progression
MH  - Drug Administration Schedule
MH  - Female
MH  - Follow-Up Studies
MH  - Humans
MH  - International Cooperation
MH  - Male
MH  - Methionine/genetics
MH  - Middle Aged
MH  - Mutation/genetics
MH  - Prealbumin/genetics/*metabolism
MH  - Quality of Life
MH  - Time Factors
MH  - Valine/genetics
MH  - Young Adult
PMC - PMC3825212
EDAT- 2013/08/27 06:00
MHDA- 2014/06/19 06:00
PMCR- 2013/08/22
CRDT- 2013/08/27 06:00
PHST- 2013/04/17 00:00 [received]
PHST- 2013/07/15 00:00 [accepted]
PHST- 2013/07/12 00:00 [revised]
PHST- 2013/08/27 06:00 [entrez]
PHST- 2013/08/27 06:00 [pubmed]
PHST- 2014/06/19 06:00 [medline]
PHST- 2013/08/22 00:00 [pmc-release]
AID - 7051 [pii]
AID - 10.1007/s00415-013-7051-7 [doi]
PST - ppublish
SO  - J Neurol. 2013 Nov;260(11):2802-14. doi: 10.1007/s00415-013-7051-7. Epub 2013 Aug 
      22.