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
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- 作者：Yukio Andoa ; andoy709@kumamoto-u.ac.jp" class="auth_mail" title="E-mail the corresponding author ; Yoshiki Sekijimab ; sekijima@shinshu-u.ac.jp" class="auth_mail" title="E-mail the corresponding author ; Konen Obayashic ; konen@kumamoto-u.ac.jp" class="auth_mail" title="E-mail the corresponding author ; Taro Yamashitaa ; taro-yamashita@fc.kuh.kumamoto-u.ac.jp" class="auth_mail" title="E-mail the corresponding author ; Mitsuharu Uedaa ; mitt@rb3.so-net.ne.jp" class="auth_mail" title="E-mail the corresponding author ; Yohei Misumia ; misumiyohei@hotmail.co.jp" class="auth_mail" title="E-mail the corresponding author ; Hiroshi Moritab ; hmorita@shinshu-u.ac.jp" class="auth_mail" title="E-mail the corresponding author ; Katsuyuki Machiid ; 1 ; kachannachan@aol.com" class="auth_mail" title="E-mail the corresponding author ; Makoto Ohtad ; makoto.ohta@pfizer.com" class="auth_mail" title="E-mail the corresponding author ; Ami Takatad ; Ami.Takata@pfizer.com" class="auth_mail" title="E-mail the corresponding author ; Shû ; -ichi Ikedab ; ikedasi@shinshu-u.ac.jp" class="auth_mail" title="E-mail the corresponding author
- 关键词：TTR ; transthyretin ; TTR-FAP ; TTR familial amyloid polyneuropathy ; mBMI ; modified body mass index ; NIS ; Neuropathy Impairment Score ; NIS-LL ; Neuropathy Impairment Score-Lower Limb ; NIS-UL ; Neuropathy Impairment Score-Upper Limb ; TQOL ; Norfolk QOL-DN total quality of life ; NSAID ; nonsteroidal anti-inflammatory drug
- 刊名：Journal of the Neurological Sciences
- 出版年：2016
- 出版时间：15 March 2016
- 年：2016
- 卷：362
- 期：Complete
- 页码：266-271
- 全文大小：492 K
文摘
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The efficacy and safety of 20 mg tafamidis in patients with TTR-FAP was evaluated.
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All 10 patients achieved TTR stabilization (primary endpoint) at Weeks 8 and 26.
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Minimal neurological progression was observed as measured by NIS-LL.
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Nutrition status (mBMI) improved over the study while TQOL was variable.
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Tafamidis slows neurological progression and improves nutrition status in TTR-FAP.