Early intervention with tafamidis provides long-term benefit in delaying neurological progression in patients with transthyretin familial amyloid polyneuropathy

被引:0
|
作者
Marcia Waddington-Cruz
Leslie Amass
Denis Keohane
Jeffrey Schwartz
Balarama Gundapaneni
Hui Hua Li
机构
[1] Neuromuscular Diseases Unit,Hospital Universitario Clementino
[2] Pfizer,undefined
[3] Pfizer,undefined
[4] Pfizer,undefined
[5] Pfizer,undefined
[6] Pfizer,undefined
[7] Pfizer,undefined
[8] InVentiv Health,undefined
关键词
Registration Trial; Open Label Extension; Additional Categorization; Continue Evaluation; Treatment Start;
D O I
10.1186/1750-1172-10-S1-P12
中图分类号
学科分类号
摘要
引用
收藏
相关论文
共 50 条
  • [31] Long-Term Effect of Tafamidis on Clinical Parameters and Prognostic Predictors in Patients With Transthyretin Amyloid Cardiomyopathy
    Kuyama, Naoto
    Izumiya, Yasuhiro
    Takashio, Seiji
    Usuku, Hiroki
    Tabira, Akihisa
    Oguni, Tetsuya
    Yamamoto, Masahiro
    Hirakawa, Kyoko
    Ishii, Masanobu
    Tabata, Noriaki
    Hoshiyama, Tadashi
    Kanazawa, Hisanori
    Hanatani, Shinsuke
    Kidoh, Masafumi
    Oda, Seitaro
    Matsuzawa, Yasushi
    Yamamoto, Eiichiro
    Hirai, Toshinori
    Ueda, Mitsuharu
    Tsujita, Kenichi
    CIRCULATION JOURNAL, 2025, 89 (04) : 421 - 431
  • [32] Transthyretin familial amyloid polyneuropathy: The neuropathy progression on treated patients compared with natural disease progression
    Miranda, B.
    Castro, J.
    Conceicao, I.
    EUROPEAN JOURNAL OF NEUROLOGY, 2017, 24 : 31 - 31
  • [33] Tafamidis delays neurological progression comparably across Val30Met and non-Val30Met genotypes in transthyretin familial amyloid polyneuropathy
    Gundapaneni, B. K.
    Sultan, M. B.
    Keohane, D. J.
    Schwartz, J. H.
    EUROPEAN JOURNAL OF NEUROLOGY, 2018, 25 (03) : 464 - 468
  • [34] Long-term tafamidis efficacy in patients with transthyretin amyloid cardiomyopathy by baseline left ventricular ejection fraction
    Drachman, Brian
    Damy, Thibaud
    Hanna, Mazen
    Wang, Ronnie
    Angeli, Franca S.
    Garcia-Pavia, Pablo
    EUROPEAN JOURNAL OF HEART FAILURE, 2024, 26 (09) : 2038 - 2046
  • [35] INFLUENCE OF BASELINE NEUROLOGIC SEVERITY ON DISEASE PROGRESSION AND THE ASSOCIATED DISEASE-MODIFYING EFFECTS OF TAFAMIDIS IN TRANSTHYRETIN FAMILIAL AMYLOID POLYNEUROPATHY
    Amass, L.
    Li, H.
    Gundapaneni, B.
    Schwartz, J.
    Keohane, D.
    JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM, 2017, 22 (03) : 235 - 235
  • [36] Long-term survival after liver transplantation in patients with familial amyloid polyneuropathy
    Yamashita, T.
    Ando, Y.
    Okamoto, S.
    Misumi, Y.
    Hirahara, T.
    Ueda, M.
    Obayashi, K.
    Nakamura, M.
    Jono, H.
    Shono, M.
    Asonuma, K.
    Inomata, Y.
    Uchino, M.
    NEUROLOGY, 2012, 78 (09) : 637 - 643
  • [37] Improved long-term survival with tafamidis treatment in patients with transthyretin amyloid cardiomyopathy and severe heart failure symptoms
    Elliott, Perry
    Gundapaneni, Balarama
    Sultan, Marla B.
    Ines, Monica
    Garcia-Pavia, Pablo
    EUROPEAN JOURNAL OF HEART FAILURE, 2023, 25 (11) : 2060 - 2064
  • [38] Letter by Baldovino et al Regarding Article, "Long-Term Survival With Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy"
    Baldovino, Simone
    Costanzo, Piera
    Roccatello, Dario
    CIRCULATION-HEART FAILURE, 2022, 15 (07)
  • [39] Interim analysis of data from a long-term, extension trial of tafamidis meglumine in patients with transthyretin amyloid cardiomyopathy
    Elliott, P.
    Drachman, B. M.
    Gottlieb, S. S.
    Hoffman, J. E.
    Hummel, S. L.
    Lenihan, D. J.
    Ebede, B.
    Gundapaneni, B.
    Schwartz, J. H.
    Sultan, M. B.
    Shah, S. J.
    EUROPEAN HEART JOURNAL, 2019, 40 : 645 - 645
  • [40] Effect of long-term tafamidis treatment on health-related quality of life in patients with transthyretin amyloid cardiomyopathy
    Grogan, Martha
    Davis, Margot K.
    Crespo-Leiro, Maria G.
    Sultan, Marla B.
    Gundapaneni, Balarama
    Angeli, Franca Stedile
    Hanna, Mazen
    EUROPEAN JOURNAL OF HEART FAILURE, 2024, 26 (03) : 612 - 615