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

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. We assessed the cost-effectiveness of universal systematic screening (USS) using PYP-scans in patients aged 60 years or older with heart failure with preserved ejection fraction (HFpEF) and ventricular wall thickness of at least 12mm. Methods Two screening strategies, USS of ATTR-CM with PYP-scan versus selected clinical referrals for PYP-scan in standard-of-care (SoC), 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 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. What is Known - ATTR-CM is a rare but serious condition that leads to a restrictive cardiomyopathy with symptoms that mimic heart failure from other causes (e.g., HFpEF). - PYP-scan, when it is used along with the other conventional diagnostic tests, is a non-invasive diagnostic tool that sensitively and specifically detect ATTR-CM followed by a timely treatment with tafamidis. - Tafamidis can slow down ATTR-CM progression and improve survival. What the Study Adds - This is the first attempt to evaluate the cost-effectiveness of universal systematic screening of ATTR-CM using PYP-scans in older HFpEF patients with ventricular wall thickening. - The study found that the universal systematic screening is not cost-effective compared to standard-of-care at a liberal WTP threshold, mainly due to the costly treatment option. - The study identified the key parameters that affect the cost-effectiveness of screening, such as age, prevalence, and drug cost.