PMID- 33895806
OWN - NLM
STAT- MEDLINE
DCOM- 20220818
LR  - 20221228
IS  - 2058-1742 (Electronic)
IS  - 2058-5225 (Print)
IS  - 2058-1742 (Linking)
VI  - 8
IP  - 5
DP  - 2022 Aug 17
TI  - Health impact of tafamidis in transthyretin amyloid cardiomyopathy patients: an 
      analysis from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial 
      (ATTR-ACT) and the open-label long-term extension studies.
PG  - 529-538
LID - 10.1093/ehjqcco/qcab031 [doi]
AB  - AIM: The Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) 
      showed that tafamidis reduced all-cause mortality and cardiovascular-related 
      hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). 
      This study aimed to estimate the impact of tafamidis on survival and 
      quality-adjusted life-years (QALYs). METHODS AND RESULTS: A multi-state, cohort, 
      Markov model was developed to simulate the disease course of ATTR-CM throughout a 
      lifetime. For survival extrapolation, survival curves were fitted by treatment 
      arm and New York Heart Association (NYHA) Class I/II (68% of patients) and NYHA 
      Class III (32% of patients) cohorts using the individual patient-level data from 
      both the ATTR-ACT and the corresponding long-term extension study. Univariate and 
      multivariate sensitivity analyses were conducted. The predicted mean survival for 
      the total population (NYHA Class I/II + III) was 6.73 years for tafamidis and 
      2.85 years for the standard of care (SoC), resulting in an incremental mean 
      survival of 3.88 years [95% confidence interval (CI) 1.32-5.66]. Of the 6.73 
      life-years, patients on tafamidis spend, on average, 4.82 years in NYHA Class 
      I/II, while patients on SoC spend an average of 1.60 life-years in these classes. 
      The combination of longer survival in lower NYHA classes produced a QALY gain of 
      5.39 for tafamidis and 2.11 for SoC, resulting in 3.29 incremental QALYs (95% CI 
      1.21-4.74) in favour of tafamidis. CONCLUSION: Based on the disease simulation 
      model results, tafamidis is expected to more than double the life expectancy and 
      QALYs of ATTR-CM patients compared to SoC. Longer-term follow-up data from the 
      ATTR-ACT extension study will further inform these findings. CLINICAL TRIALS.GOV 
      IDENTIFIER: NCT01994889 (date of registration: 26 November 2013).
CI  - (c) The Author(s) 2021. Published by Oxford University Press on behalf of the 
      European Society of Cardiology.
FAU - Rozenbaum, Mark H
AU  - Rozenbaum MH
AD  - Pfizer Inc., Rivium Westlaan 142, 2909 LD, Capelle aan den IJssel, The 
      Netherlands.
FAU - Garcia, Andrea
AU  - Garcia A
AUID- ORCID: 0000-0003-4388-2608
AD  - Ingress-health, Weena 316-318 3012 NJ, Rotterdam, The Netherlands.
FAU - Grima, Daniel
AU  - Grima D
AD  - Eversana Life Science Services, 204-3228 South Service Road, Burlington L7N 3H8 
      ON, Canada.
FAU - Tran, Diana
AU  - Tran D
AD  - Eversana Life Science Services, 204-3228 South Service Road, Burlington L7N 3H8 
      ON, Canada.
FAU - Bhambri, Rahul
AU  - Bhambri R
AD  - Pfizer Inc., 235 E 42nd St, New York, NY, USA.
FAU - Stewart, Michelle
AU  - Stewart M
AD  - Pfizer Inc., 235 E 42nd St, New York, NY, USA.
FAU - Li, Benjamin
AU  - Li B
AD  - Pfizer Inc., 235 E 42nd St, New York, NY, USA.
FAU - Heeg, Bart
AU  - Heeg B
AD  - Ingress-health, Weena 316-318 3012 NJ, Rotterdam, The Netherlands.
FAU - Postma, Maarten
AU  - Postma M
AD  - Unit of PharmacoEpidemiology & PharmacoEconomics, Department of Pharmacy, 
      University of Groningen, Antonius Deusinglaan 1, 9713 AV Groningen, The 
      Netherlands.
AD  - Unit of Economics, Econometrics & Finance, Faculty of Economics & Business, 
      University of Groningen, Nettelbosje 2, 9747 AE, Groningen, The Netherlands.
AD  - Department of Health Sciences, University of Groningen, University Medical Center 
      Groningen, Antonius Deusinglaan 1, 9713 AV Groningen NL, The Netherlands.
FAU - Masri, Ahmad
AU  - Masri A
AUID- ORCID: 0000-0002-6390-6526
AD  - The Amyloidosis Center, Knight Cardiovascular Institute, Oregon Health & Science 
      University, 3303 S Bond Ave Building 1, 9th Floor, Portland, OR 97239, USA.
LA  - eng
SI  - ClinicalTrials.gov/NCT01994889
PT  - Clinical Trial
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PL  - England
TA  - Eur Heart J Qual Care Clin Outcomes
JT  - European heart journal. Quality of care & clinical outcomes
JID - 101677796
RN  - 0 (Benzoxazoles)
RN  - 0 (Prealbumin)
RN  - 8FG9H9D31J (tafamidis)
SB  - IM
MH  - *Amyloid Neuropathies, Familial/complications/drug therapy
MH  - Benzoxazoles/therapeutic use
MH  - *Cardiomyopathies/complications/drug therapy
MH  - Humans
MH  - Prealbumin/therapeutic use
PMC - PMC9382662
OTO - NOTNLM
OT  - Cardiomyopathy
OT  - Mortality
OT  - Tafamidis
OT  - Transthyretin
OT  -  Amyloidosis
EDAT- 2021/04/26 06:00
MHDA- 2022/08/19 06:00
PMCR- 2021/04/24
CRDT- 2021/04/25 20:51
PHST- 2021/01/21 00:00 [received]
PHST- 2021/04/16 00:00 [revised]
PHST- 2021/04/21 00:00 [accepted]
PHST- 2021/04/26 06:00 [pubmed]
PHST- 2022/08/19 06:00 [medline]
PHST- 2021/04/25 20:51 [entrez]
PHST- 2021/04/24 00:00 [pmc-release]
AID - 6251357 [pii]
AID - qcab031 [pii]
AID - 10.1093/ehjqcco/qcab031 [doi]
PST - ppublish
SO  - Eur Heart J Qual Care Clin Outcomes. 2022 Aug 17;8(5):529-538. doi: 
      10.1093/ehjqcco/qcab031.