PMID- 37434378
OWN - NLM
STAT- MEDLINE
DCOM- 20231216
LR  - 20231218
IS  - 1879-0844 (Electronic)
IS  - 1388-9842 (Linking)
VI  - 25
IP  - 11
DP  - 2023 Nov
TI  - Improved long-term survival with tafamidis treatment in patients with 
      transthyretin amyloid cardiomyopathy and severe heart failure symptoms.
PG  - 2060-2064
LID - 10.1002/ejhf.2974 [doi]
AB  - AIM: The value of disease-modifying therapies (such as tafamidis) in patients 
      with transthyretin amyloid cardiomyopathy (ATTR-CM) and severe heart failure 
      symptoms has been debated. This study assessed long-term all-cause survival in 
      patients with New York Heart Association (NYHA) class III symptoms in the 
      Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) long-term 
      extension (LTE) study. METHODS AND RESULTS: At the baseline of ATTR-ACT, 55/176 
      (31.3%) patients receiving tafamidis 80 mg and 63/177 (35.6%) receiving placebo 
      had NYHA class III symptoms. After 30 months of treatment, patients could join an 
      ongoing LTE study to receive open-label tafamidis. In an interim analysis of the 
      LTE study (August 2021), all-cause mortality was lower among patients with NYHA 
      class III symptoms who received continuous tafamidis in ATTR-ACT and the LTE 
      study (hazard ratio 0.64; 95% confidence interval 0.41-0.99; median follow-up: 
      60 months), as compared with those who received placebo in ATTR-ACT and tafamidis 
      in the LTE study (median follow-up: 56 months). Similar findings were observed in 
      patients with NYHA class I/II symptoms at baseline (0.50; 0.35-0.73; tafamidis 
      80 mg n = 121; placebo n = 114; median follow-up of 61 and 60 months, 
      respectively). CONCLUSION: We observed reduced all-cause mortality with 
      continuous tafamidis treatment compared with delayed tafamidis treatment (placebo 
      then tafamidis) in patients with NYHA class III symptoms at baseline over a 
      median follow-up of  approximately 5 years. These findings demonstrate the value of tafamidis 
      treatment in patients with ATTR-CM and severe heart failure symptoms, and 
      emphasize the importance of early treatment. CLINICAL TRIAL REGISTRATIONS: 
      ClinicalTrials.gov NCT01994889 and NCT02791230.
CI  - (c) 2023 Pfizer Inc and The Authors. European Journal of Heart Failure published by 
      John Wiley & Sons Ltd on behalf of European Society of Cardiology.
FAU - Elliott, Perry
AU  - Elliott P
AD  - University College London, London, UK.
FAU - Gundapaneni, Balarama
AU  - Gundapaneni B
AD  - Pfizer Inc, Groton, CT, USA.
FAU - Sultan, Marla B
AU  - Sultan MB
AD  - Pfizer Inc, New York, NY, USA.
FAU - Ines, Monica
AU  - Ines M
AD  - Pfizer Inc, Porto Salvo, Portugal.
FAU - Garcia-Pavia, Pablo
AU  - Garcia-Pavia P
AD  - Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV, Madrid, 
      Spain.
AD  - Universidad Francisco de Vitoria, Madrid, Spain.
AD  - CNIC, Madrid, Spain.
LA  - eng
SI  - ClinicalTrials.gov/NCT02791230
SI  - ClinicalTrials.gov/NCT01994889
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20230726
PL  - England
TA  - Eur J Heart Fail
JT  - European journal of heart failure
JID - 100887595
RN  - 8FG9H9D31J (tafamidis)
RN  - 0 (Prealbumin)
SB  - IM
CIN - Eur J Heart Fail. 2023 Nov;25(11):2065-2066. PMID: 37792736
MH  - Humans
MH  - *Amyloid Neuropathies, Familial/complications/drug therapy
MH  - Prealbumin
MH  - *Heart Failure/drug therapy/complications
MH  - *Cardiomyopathies/drug therapy/complications
OTO - NOTNLM
OT  - ATTR-ACT
OT  - Amyloidosis
OT  - Prognosis
OT  - Survival rate
OT  - Trial
EDAT- 2023/07/12 06:42
MHDA- 2023/12/17 09:45
CRDT- 2023/07/12 02:04
PHST- 2023/06/30 00:00 [revised]
PHST- 2022/10/14 00:00 [received]
PHST- 2023/07/09 00:00 [accepted]
PHST- 2023/12/17 09:45 [medline]
PHST- 2023/07/12 06:42 [pubmed]
PHST- 2023/07/12 02:04 [entrez]
AID - 10.1002/ejhf.2974 [doi]
PST - ppublish
SO  - Eur J Heart Fail. 2023 Nov;25(11):2060-2064. doi: 10.1002/ejhf.2974. Epub 2023 
      Jul 26.