TY - JOUR
T1 - Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis
AU - Merlini, Giampaolo
AU - Planté-Bordeneuve, Violaine
AU - Judge, Daniel P.
AU - Schmidt, Hartmut
AU - Obici, Laura
AU - Perlini, Stefano
AU - Packman, Jeff
AU - Tripp, Tara
AU - Grogan, Donna R.
PY - 2013/12
Y1 - 2013/12
N2 - 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.
AB - 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.
KW - Cardiomyopathy
KW - Familial amyloid polyneuropathy
KW - Tafamidis
KW - Transthyretin amyloidosis
UR - http://www.scopus.com/inward/record.url?scp=84890488396&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84890488396&partnerID=8YFLogxK
U2 - 10.1007/s12265-013-9512-x
DO - 10.1007/s12265-013-9512-x
M3 - Article
C2 - 24101373
AN - SCOPUS:84890488396
SN - 1937-5387
VL - 6
SP - 1011
EP - 1020
JO - Journal of Cardiovascular Translational Research
JF - Journal of Cardiovascular Translational Research
IS - 6
ER -