Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy

Autor: Richard Labaudinière, Pedro Trigo, Isabel Conceição, Jeffery W. Kelly, Ana Martins da Silva, Jason L. Chan, Donna R. Grogan, Jeff Packman, Ole B. Suhr, Márcia Waddington Cruz, Teresa Coelho, Hartmut Schmidt, Luis F. Maia, Violaine Planté-Bordeneuve
Rok vydání: 2013
Předmět:
Male
Tafamidis
Time Factors
International Cooperation
030204 cardiovascular system & hematology
Transthyretin amyloidosis
law.invention
chemistry.chemical_compound
Methionine
0302 clinical medicine
Quality of life
Randomized controlled trial
law
Disease modification
Prealbumin
Benzoxazoles
Original Communication
biology
Valine
Middle Aged
3. Good health
Neurology
Tolerability
Disease Progression
Female
Adult
medicine.medical_specialty
Clinical Neurology
Placebo
Drug Administration Schedule
Young Adult
03 medical and health sciences
Internal medicine
medicine
Humans
Adverse effect
Aged
Amyloid Neuropathies
Familial
business.industry
Surgery
Amyloid Neuropathy
Transthyretin
chemistry
Familial amyloid polyneuropathy
Mutation
Quality of Life
biology.protein
Neurology (clinical)
business
030217 neurology & neurosurgery
Follow-Up Studies
Zdroj: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Agência para a Sociedade do Conhecimento (UMIC)-FCT-Sociedade da Informação
instacron:RCAAP
Journal of Neurology
ISSN: 1432-1459
0340-5354
DOI: 10.1007/s00415-013-7051-7
Popis: 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.
Databáze: OpenAIRE
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