What is the role of tafamidis in the treatment of amyloidosis?

Updated: May 09, 2019
  • Author: Robert O Holmes, Jr, DO; Chief Editor: Herbert S Diamond, MD  more...
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Transthyretin amyloidosis is related to abnormal conformational changes and transthyretin aggregation, leading to severe neuropathy. After learning the structure and function of transthyretin, researchers developed a molecule and later a drug, tafamidis, that could stabilize the native tetrameric state of transthyretin. [96]

A phase II, open-label, single-treatment arm evaluated the pharmacodynamics, efficacy, and safety of tafamidis in patients with non-Val30Met transthyretin (TTR) amyloidosis. Twenty-one patients with 8 different non-Val30Met mutations received daily oral tafamidis, and many experienced improved quality of life measures, while pro-BNP and echocardiographic features improved. [98] Another study showed that long-term use continued to stabilize TTR at 30 months. In a cross-over scheme, patients previously have given placebo were able to slow their neurologic decline, proving that earlier treatment is better. [99] The success of tafamidis in a subset of amyloidosis is very encouraging for future molecular targets.

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