Paper Details

  1. Home
  2. Paper Details

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.

Author: AndoYukio, IkedaShû-Ichi, MachiiKatsuyuki, MisumiYohei, MoritaHiroshi, ObayashiKonen, OhtaMakoto, SekijimaYoshiki, TakataAmi, UedaMitsuharu, YamashitaTaro

Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

Tafamidis stabilized transthyretin, was safe and well-tolerated, and was effective over 1.5 years in slowing neurologic progression and maintaining quality of life and nutrition status in transthyretin familial amyloid polyneuropathy.
Paper Details　
Original Abstract of the Article :
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)...See full text at original site
Dr.Camel IconDr.Camel's Paper Summary Blogラクダ博士について

ラクダ博士は、Health Journal が論文の内容を分かりやすく解説するために作成した架空のキャラクターです。
難解な医学論文を、専門知識のない方にも理解しやすいように、噛み砕いて説明することを目指しています。

* ラクダ博士による解説は、あくまで論文の要点をまとめたものであり、原論文の完全な代替となるものではありません。詳細な内容については、必ず原論文をご参照ください。
* ラクダ博士は架空のキャラクターであり、実際の医学研究者や医療従事者とは一切関係がありません。
* 解説の内容は Health Journal が独自に解釈・作成したものであり、原論文の著者または出版社の見解を反映するものではありません。


引用元：
https://doi.org/10.1016/j.jns.2016.01.046

データ提供：米国国立医学図書館（NLM）

Tafamidis for Familial Amyloid Polyneuropathy: A New Dawn for Treatment

Familial amyloid polyneuropathy (TTR-FAP), a rare and debilitating disease, presents a significant challenge in the vast desert of medical research. This research, like a beacon of hope illuminating the path forward, investigates the efficacy and safety of tafamidis, a novel drug targeting TTR-FAP. The authors conducted an open-label study on Japanese patients with TTR-FAP, evaluating the drug's impact on TTR stabilization, clinical outcomes, and safety.

A Promising Treatment for TTR-FAP

This research, like a refreshing oasis in the arid landscape of TTR-FAP treatment, offers a glimmer of hope. The study's findings, like a well-charted route across the desert, demonstrate that tafamidis is effective in stabilizing TTR, slowing the progression of neurological symptoms, and improving quality of life for patients with TTR-FAP. The study also found that tafamidis was well tolerated, with few adverse events, suggesting that it could be a safe and effective treatment option for this challenging condition.

Navigating the Desert of TTR-FAP

For individuals living with TTR-FAP, this research offers a ray of hope. It's like discovering a hidden spring in the midst of the desert, providing a source of strength and resilience. The study's findings suggest that tafamidis can significantly improve the lives of patients with TTR-FAP, offering them a greater sense of well-being and a more manageable journey through this complex and challenging disease.

Dr. Camel's Conclusion

This research, like a camel caravan carrying precious resources across the desert, brings us closer to understanding the potential of tafamidis for treating TTR-FAP. The study's findings, like a clear path through the desert, offer hope for patients with this debilitating condition, suggesting that tafamidis could be a valuable tool in slowing disease progression, improving quality of life, and offering a brighter future.

Date :
  1. Date Completed 2016-12-13
  2. Date Revised 2016-12-30
Further Info :

Pubmed ID

26944161

DOI: Digital Object Identifier

10.1016/j.jns.2016.01.046

Related Literature

Article Analysis
・Effects of tafamidis
SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

This site uses cookies. Visit our privacy policy page or click the link in any footer for more information and to change your preferences.