PMID- 37979789
OWN - NLM
STAT- MEDLINE
DCOM- 20240123
LR  - 20240131
IS  - 1874-1754 (Electronic)
IS  - 0167-5273 (Linking)
VI  - 398
DP  - 2024 Mar 1
TI  - Cost-effectiveness of systematic screening and treatment of transthyretin amyloid 
      cardiomyopathy (ATTR-CM) in patients with heart failure with preserved ejection 
      fraction (HFpEF) in United States.
PG  - 131598
LID - S0167-5273(23)01688-1 [pii]
LID - 10.1016/j.ijcard.2023.131598 [doi]
AB  - BACKGROUND: Transthyretin amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed 
      cause of heart failure in clinical practice. (99m)Tc-pyrophosphate scintigraphy 
      (PYP-scan) improves the accuracy of ATTR-CM detection, enabling timely initiation 
      of tafamidis, a drug that slows the progression of ATTR-CM and lowers the risk of 
      adverse cardiac events. PYP-scans, serum free light-chain (FLC) test and 
      immunofixation electrophoresis (IFE) are critical components of a systematic 
      screening. We assessed the cost-effectiveness of universal systematic screening 
      (USS) compared to standard-of-care (SoC) selected clinical referrals for the 
      systematic screening in patients aged 60 years or older with heart failure with 
      preserved ejection fraction (HFpEF) and ventricular wall thickness of at least 
      12 mm. METHODS: Two screening strategies, USS versus SoC screening for ATTR-CM 
      were compared in a model-based assessment. Treatment decisions were based upon 
      the accuracy of each screening strategy, which was followed by Markov state 
      transitions across New York Heart Association (NYHA) functional classes and 
      death. Model inputs were identified from a literature review. We calculated 
      lifetime cost in 2022 US dollars and quality adjusted life-years (QALYs) of each 
      strategy. The primary outcome was the incremental cost-effectiveness ratio 
      (ICER). RESULTS: The USS was associated with a significant increase in lifetime 
      costs ($124,380 vs. $70,412) and modest improvement in QALYs (4.42 QALYs vs 4.36 
      QALYs). The ICER for the USS was $919,509 per QALY gained. ICER was sensitive to 
      the age at the time of ATTR-CM diagnosis, true prevalence rate of ATTR-CM, and 
      daily cost of tafamidis. CONCLUSIONS: Owing to the high cost of treatment with 
      tafamidis, USS along with PYP scan for ATTR-CM in older HFpEF patients with 
      ventricular wall thickening is unlikely to become a cost-effective strategy at a 
      liberal WTP threshold.
CI  - Copyright (c) 2023 Elsevier B.V. All rights reserved.
FAU - Lau, Anson T C
AU  - Lau ATC
AD  - Department of Pharmacy Systems, Outcomes and Policy, University of Illinois 
      Chicago, Chicago, IL, United States of America.
FAU - DiDomenico, Robert J
AU  - DiDomenico RJ
AD  - Department of Pharmacy Practice, University of Illinois Chicago, Chicago, IL, 
      United States of America; Center for Pharmacoepidemiology and Pharmacoeconomic 
      Research, University of Illinois Chicago, Chicago, IL, United States of America.
FAU - Kim, Kibum
AU  - Kim K
AD  - Department of Pharmacy Systems, Outcomes and Policy, University of Illinois 
      Chicago, Chicago, IL, United States of America; Center for Pharmacoepidemiology 
      and Pharmacoeconomic Research, University of Illinois Chicago, Chicago, IL, 
      United States of America. Electronic address: kkim204@uic.edu.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20231117
PL  - Netherlands
TA  - Int J Cardiol
JT  - International journal of cardiology
JID - 8200291
RN  - 0 (Prealbumin)
SB  - IM
MH  - Humans
MH  - United States/epidemiology
MH  - Aged
MH  - Cost-Benefit Analysis
MH  - Prealbumin
MH  - *Heart Failure/diagnostic imaging/drug therapy
MH  - Stroke Volume
MH  - *Amyloidosis
MH  - *Cardiomyopathies/diagnostic imaging/drug therapy
MH  - *Amyloid Neuropathies, Familial/diagnostic imaging/drug therapy
OTO - NOTNLM
OT  - Cost-effectiveness analysis
OT  - Heart failure with preserved ejection fraction
OT  - Tafamidis
OT  - Technetium Tc 99 m pyrophosphate scintigraphy
OT  - Transthyretin amyloid cardiomyopathy
COIS- Declaration of Competing Interest Anson Lau serves as a Senior Medical Advisor in 
      AstraZeneca. Kibum Kim has received research funding from Renalytix AI, Grail LLC 
      (a subsidiary of Illumina Inc.), and AstraZeneca. Robert DiDomenico has received 
      research funding from the Chicago Department of Public Health, Cook County 
      Department of Public Health, Otho S.A. Sprague Memorial Institute, and CSL 
      Behring and has served on advisory boards for Abiomed and PhaseBio 
      Pharmaceuticals.
EDAT- 2023/11/19 09:42
MHDA- 2024/01/23 06:42
CRDT- 2023/11/18 19:30
PHST- 2023/09/06 00:00 [received]
PHST- 2023/10/25 00:00 [revised]
PHST- 2023/11/14 00:00 [accepted]
PHST- 2024/01/23 06:42 [medline]
PHST- 2023/11/19 09:42 [pubmed]
PHST- 2023/11/18 19:30 [entrez]
AID - S0167-5273(23)01688-1 [pii]
AID - 10.1016/j.ijcard.2023.131598 [doi]
PST - ppublish
SO  - Int J Cardiol. 2024 Mar 1;398:131598. doi: 10.1016/j.ijcard.2023.131598. Epub 
      2023 Nov 17.