Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis

Giampaolo Merlini, Violaine Planté-Bordeneuve, Daniel P. Judge, Hartmut Schmidt, Laura Obici, Stefano Perlini, Jeff Packman, Tara Tripp, Donna R. Grogan

Research output: Contribution to journalArticle

Abstract

This phase II, open-label, single-treatment arm study evaluated the pharmacodynamics, efficacy, and safety of tafamidis in patients with non-Val30Met transthyretin (TTR) amyloidosis. Twenty-one patients with eight different non-Val30Met mutations received 20 mg QD of tafamidis meglumine for 12 months. The primary outcome, TTR stabilization at Week 6, was achieved in 18 (94.7 %) of 19 patients with evaluable data. TTR was stabilized in 100 % of patients with non-missing data at Months 6 (n = 18) and 12 (n = 17). Exploratory efficacy measures demonstrated some worsening of neurological function. However, health-related quality of life, cardiac biomarker N-terminal pro-hormone brain natriuretic peptide, echocardiographic parameters, and modified body mass index did not demonstrate clinically relevant worsening during the 12 months of treatment. Tafamidis was well tolerated. In conclusion, our findings suggest that tafamidis 20 mg QD effectively stabilized TTR associated with several non-Val30Met variants.

Original languageEnglish
Pages (from-to)1011-1020
Number of pages10
JournalJournal of Cardiovascular Translational Research
Volume6
Issue number6
DOIs
Publication statusPublished - Dec 2013

Fingerprint

Prealbumin
Brain Natriuretic Peptide
Body Mass Index
Biomarkers
Quality of Life
Hormones
Safety
Mutation
Amyloidosis, Hereditary, Transthyretin-Related
tafamidis
Therapeutics

Keywords

  • Cardiomyopathy
  • Familial amyloid polyneuropathy
  • Tafamidis
  • Transthyretin amyloidosis

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine
  • Genetics
  • Genetics(clinical)
  • Molecular Medicine
  • Pharmaceutical Science

Cite this

Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis. / Merlini, Giampaolo; Planté-Bordeneuve, Violaine; Judge, Daniel P.; Schmidt, Hartmut; Obici, Laura; Perlini, Stefano; Packman, Jeff; Tripp, Tara; Grogan, Donna R.

In: Journal of Cardiovascular Translational Research, Vol. 6, No. 6, 12.2013, p. 1011-1020.

Research output: Contribution to journalArticle

Merlini, Giampaolo ; Planté-Bordeneuve, Violaine ; Judge, Daniel P. ; Schmidt, Hartmut ; Obici, Laura ; Perlini, Stefano ; Packman, Jeff ; Tripp, Tara ; Grogan, Donna R. / Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis. In: Journal of Cardiovascular Translational Research. 2013 ; Vol. 6, No. 6. pp. 1011-1020.
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