PMID- 26944161
OWN - NLM
STAT- MEDLINE
DCOM- 20161213
LR  - 20161230
IS  - 1878-5883 (Electronic)
IS  - 0022-510X (Linking)
VI  - 362
DP  - 2016 Mar 15
TI  - Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, 
      and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) 
      with Val30Met and non-Val30Met: A phase III, open-label study.
PG  - 266-71
LID - S0022-510X(16)30046-6 [pii]
LID - 10.1016/j.jns.2016.01.046 [doi]
AB  - INTRODUCTION: The efficacy and safety of tafamidis in transthyretin (TTR) 
      familial amyloid polyneuropathy (TTR-FAP) were evaluated in this open-label 
      study. METHODS: Japanese TTR-FAP patients (n=10; mean age 60.1 years) received 
      tafamidis meglumine (20mg daily; median treatment duration 713.5 days). The 
      primary endpoint was TTR stabilization at Week 8. Secondary endpoints included 
      Neuropathy Impairment Score-Lower Limb (NIS-LL), Norfolk QOL-DN total quality of 
      life (TQOL), and modified body mass index (mBMI). RESULTS: TTR stabilization was 
      achieved in all patients at Weeks 8 and 26, 9 out of 10 patients at Week 52, and 
      8 out of 10 patients at Week 78. The percentage (95% CI) of NIS-LL responders 
      (increase from baseline in NIS-LL<2) was 80.0% (44.4, 97.5), 60.0% (26.2, 87.8), 
      and 40.0% (12.2, 73.8) and mean(SD) NIS-LL change from baseline was 2.1 (5.6), 
      3.6 (4.4), and 3.3 (4.7), at Weeks 26, 52, and 78, respectively. Mean (SD) 
      changes from baseline in TQOL and mBMI at Weeks 26, 52, and 78 were 11.8 (20.0), 
      9.1 (12.5), and 10.8 (13.7) for TQOL, and 26.6 (61.9), 64.9 (80.0), and 53.7 
      (81.4) for mBMI, respectively. Ambulation status was preserved in 4 out of 8 
      patients at Week 78. Most adverse events (AEs) were mild/moderate, with no 
      discontinuations due to AEs. CONCLUSIONS: Tafamidis stabilized TTR, was safe and 
      well-tolerated, and was effective over 1.5 years in slowing neurologic 
      progression and maintaining TQOL and nutrition status in TTR-FAP.
CI  - Copyright (c) 2016. Published by Elsevier B.V.
FAU - Ando, Yukio
AU  - Ando Y
AD  - Department of Neurology, Graduate School of Medical Sciences, Kumamoto 
      University, Kumamoto, Japan. andoy709@kumamoto-u.ac.jp.
FAU - Sekijima, Yoshiki
AU  - Sekijima Y
AD  - Department of Medicine (Neurology and Rheumatology), Shinshu University School of 
      Medicine, Matsumoto, Japan. sekijima@shinshu-u.ac.jp.
FAU - Obayashi, Konen
AU  - Obayashi K
AD  - Department of Morphological and Physiological Sciences, Graduate School of Health 
      Sciences, Kumamoto University, Kumamoto, Japan. konen@kumamoto-u.ac.jp.
FAU - Yamashita, Taro
AU  - Yamashita T
AD  - Department of Neurology, Graduate School of Medical Sciences, Kumamoto 
      University, Kumamoto, Japan. taro-yamashita@fc.kuh.kumamoto-u.ac.jp.
FAU - Ueda, Mitsuharu
AU  - Ueda M
AD  - Department of Neurology, Graduate School of Medical Sciences, Kumamoto 
      University, Kumamoto, Japan. mitt@rb3.so-net.ne.jp.
FAU - Misumi, Yohei
AU  - Misumi Y
AD  - Department of Neurology, Graduate School of Medical Sciences, Kumamoto 
      University, Kumamoto, Japan. misumiyohei@hotmail.co.jp.
FAU - Morita, Hiroshi
AU  - Morita H
AD  - Department of Medicine (Neurology and Rheumatology), Shinshu University School of 
      Medicine, Matsumoto, Japan. hmorita@shinshu-u.ac.jp.
FAU - Machii, Katsuyuki
AU  - Machii K
AD  - Pfizer Japan Inc., Tokyo, Japan. kachannachan@aol.com.
FAU - Ohta, Makoto
AU  - Ohta M
AD  - Pfizer Japan Inc., Tokyo, Japan. makoto.ohta@pfizer.com.
FAU - Takata, Ami
AU  - Takata A
AD  - Pfizer Japan Inc., Tokyo, Japan. Ami.Takata@pfizer.com.
FAU - Ikeda, Shu-Ichi
AU  - Ikeda S
AD  - Department of Medicine (Neurology and Rheumatology), Shinshu University School of 
      Medicine, Matsumoto, Japan. ikedasi@shinshu-u.ac.jp.
LA  - eng
PT  - Clinical Trial, Phase III
PT  - Journal Article
PT  - Multicenter Study
PT  - Research Support, Non-U.S. Gov't
DEP - 20160122
PL  - Netherlands
TA  - J Neurol Sci
JT  - Journal of the neurological sciences
JID - 0375403
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
MH  - Benzoxazoles/*therapeutic use
MH  - Body Mass Index
MH  - Echocardiography
MH  - Female
MH  - Follow-Up Studies
MH  - Humans
MH  - Japan
MH  - Male
MH  - Methionine/genetics
MH  - Middle Aged
MH  - Pharmacogenetics
MH  - Prealbumin/*genetics/*metabolism
MH  - Valine/genetics
MH  - Young Adult
OTO - NOTNLM
OT  - Amyloid
OT  - TTR Val30Met
OT  - TTR non-Val30Met
OT  - familial amyloid polyneuropathy
OT  - tafamidis
OT  - transthyretin amyloidosis
EDAT- 2016/03/06 06:00
MHDA- 2016/12/15 06:00
CRDT- 2016/03/06 06:00
PHST- 2015/08/11 00:00 [received]
PHST- 2016/01/18 00:00 [revised]
PHST- 2016/01/21 00:00 [accepted]
PHST- 2016/03/06 06:00 [entrez]
PHST- 2016/03/06 06:00 [pubmed]
PHST- 2016/12/15 06:00 [medline]
AID - S0022-510X(16)30046-6 [pii]
AID - 10.1016/j.jns.2016.01.046 [doi]
PST - ppublish
SO  - J Neurol Sci. 2016 Mar 15;362:266-71. doi: 10.1016/j.jns.2016.01.046. Epub 2016 
      Jan 22.