| Title |
Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy
|
|---|---|
| Published in |
Journal of Neurology, August 2013
|
| DOI | 10.1007/s00415-013-7051-7 |
| Pubmed ID | |
| Authors |
Teresa Coelho, Luis F. Maia, Ana Martins da Silva, Márcia W. Cruz, Violaine Planté-Bordeneuve, Ole B. Suhr, Isabel Conceiçao, Hartmut H.-J. Schmidt, Pedro Trigo, Jeffery W. Kelly, Richard Labaudinière, Jason Chan, Jeff Packman, Donna R. Grogan |
| Abstract |
Tafamidis, a transthyretin (TTR) kinetic stabilizer, delayed neuropathic progression in patients with Val30Met TTR familial amyloid polyneuropathy (TTR-FAP) in an 18-month randomized controlled trial (study Fx-005). This 12-month, open-label extension study evaluated the long-term safety, tolerability, and efficacy of tafamidis 20 mg once daily in 86 patients who earlier received blinded treatment with tafamidis or placebo. Efficacy measures included the Neuropathy Impairment Score in the Lower Limbs (NIS-LL), Norfolk Quality of Life-Diabetic Neuropathy total quality of life (TQOL) score, and changes in neurologic function and nutritional status. We quantified the monthly rates of change in efficacy measures, and TTR stabilization, and monitored adverse events (AEs). Patients who continued on tafamidis had stable rates of change in NIS-LL (from 0.08 to 0.11/month; p = 0.60) and TQOL (from -0.03 to 0.25; p = 0.16). In patients switched from placebo, the monthly rate of change in NIS-LL declined (from 0.34 to 0.16/month; p = 0.01), as did TQOL score (from 0.61 to -0.16; p < 0.001). Patients treated with tafamidis for 30 months had 55.9 % greater preservation of neurologic function as measured by the NIS-LL than patients in whom tafamidis was initiated later. Plasma TTR was stabilized in 94.1 % of patients treated with tafamidis for 30 months. AEs were similar between groups; no patients discontinued because of an AE. Long-term tafamidis was well tolerated, with the reduced rate of neurologic deterioration sustained over 30 months. Tafamidis also slowed neurologic impairment in patients previously given placebo, but treatment benefits were greater when tafamidis was begun earlier. |
X Demographics
The data shown below were collected from the profile of 1 X user who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 1 | 100% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 1 | 100% |
Mendeley readers
The data shown below were compiled from readership statistics for 228 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United States | 2 | <1% |
| Israel | 1 | <1% |
| Portugal | 1 | <1% |
| Brazil | 1 | <1% |
| Unknown | 223 | 98% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Researcher | 36 | 16% |
| Student > Ph. D. Student | 29 | 13% |
| Other | 26 | 11% |
| Student > Master | 21 | 9% |
| Student > Bachelor | 20 | 9% |
| Other | 37 | 16% |
| Unknown | 59 | 26% |
| Readers by discipline | Count | As % |
|---|---|---|
| Medicine and Dentistry | 58 | 25% |
| Agricultural and Biological Sciences | 18 | 8% |
| Biochemistry, Genetics and Molecular Biology | 17 | 7% |
| Chemistry | 11 | 5% |
| Neuroscience | 11 | 5% |
| Other | 43 | 19% |
| Unknown | 70 | 31% |
Attention Score in Context
This research output has an Altmetric Attention Score of 126. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 02 April 2024.
All research outputs
#330,498
of 25,391,471 outputs
Outputs from Journal of Neurology
#34
of 4,958 outputs
Outputs of similar age
#2,367
of 210,751 outputs
Outputs of similar age from Journal of Neurology
#2
of 49 outputs
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