Table · dataset · 2026
Supplementary material: A trial emulation study indirectly comparing tafamidis and acoramidis in transthyretin amyloid cardiomyopathy: the ReplicATTR study
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<p dir="ltr"><b>These are peer-reviewed supplementary materials for the article</b><b> </b><b>'</b><b>A trial emulation study indirectly comparing tafamidis and acoramidis in transthyretin amyloid cardiomyopathy: the ReplicATTR study</b><b>'</b><b> </b><b>published in the</b><b> </b><b><i>Journal of Comparative Effectiveness Research.</i></b></p><ul><li><b>Supplemental figure 1: </b>Pictorial representation of the trial emulation study comparing tafamidis and acoramidis in transthyretin amyloid cardiomyopathy using calibration and indirect treatment comparison methods</li><li><b>Supplemental table 1:</b> Exclusion criteria to align the real-world tafamidis cohort with ATTR-ACT</li><li><b>Supplemental table 2: </b>Exclusion criteria to align the real-world tafamidis cohort with ATTRibute-CM</li><li><b>Supplemental table 3:</b> Attrition for the real-world tafamidis cohort using ATTR-ACT criteria</li><li><b>Supplemental table 4:</b> Attrition for the real-world tafamidis cohort using ATTRibute-CM criteria</li><li><b>Supplemental table 5: </b>Distributions used for the Monte Carlo simulation</li><li><b>Supplemental table 6:</b> Pre- and post-ATTR-ACT trial-emulated real-world cohort characteristics compared to ATTR-ACT trial aggregate data</li><li><b>Supplemental table 7:</b> Pre- and post-ATTRibute-CM trial-emulated real-world cohort characteristics compared to ATTRibute-CM trial aggregate data</li><li><b>Supplemental table 8:</b> Baseline characteristics of patients included in the (pre-MAIC) ATTRibute-CM-like tafamidis cohort compared to patients excluded from the cohort due to missing NT-proBNP data</li><li><b>References</b></li></ul><p dir="ltr"><b>Aim:</b> Tafamidis and acoramidis are transthyretin stabilizers approved for the treatment of transthyretin amyloid cardiomyopathy.
We compared the effectiveness of acoramidis versus tafamidis regarding allcause mortality (ACM). <b>Materials & methods:</b> This study used a hybrid design to compare 42-month ACM with acoramidis versus tafamidis using trial emulation, calibration, and indirect treatment comparison. Two real-world cohorts of participants initiating tafamidis were identified from TriNetX (2019–2024) using observational analogues to emulate ATTR-ACT and ATTRibute-CM trial eligibility criteria and baseline characteristics.
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A calibration factor was derived by comparing 42-month ACM in the ATTR-ACT tafamidis arm with the ATTR-ACT emulation cohort to quantify the net effect of systematic differences between the populations. This factor was applied to the ATTRibute-CM emulation tafamidis cohort and compared with the ATTRibute-CM acoramidis arm. Using these values and corresponding variance, Monte Carlo simulation (MCS)-estimated medians and percentile-based 95% CIs were derived. <b>Results:</b> In the ATTRibute-CM emulation tafamidis cohort (n = 617), 42-month ACM risk was 35.4% (95% CI: 29.6–42.0), and decreased to aMCS-estimatedmedian of 31.2% (95% CI: 25.3–39.0) after application of the calibration factor (MCS-estimated median: 1.14 [95% CI: 0.91–1.43]).
Comparison with 42-month ACM risk observed in the ATTRibute-CM acoramidis arm (24.0%, 95% CI: 20.0–28.7) yielded a relative risk of 0.78 (95% CI: 0.60–0.99), consistent with lower observed ACM risk in the acoramidis arm. <b>Conclusion: </b>Acoramidis was linked to a lower estimated ACM risk in this emulated comparative analysis. These findings should be considered as hypothesis-generating, requiring confirmation in head-to-head studies.</p>
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Topics
- From keywords
- Astronomy & Astrophysics · Chemistry · Computer Science & AI · Earth & Environmental Science · Economics & Finance · Engineering · Humanities · Life Sciences · Medicine & Health · Ocean & Atmospheric Science · Social Science
- Inferred from text
- Cardiomyopathy 75% · Medical biochemistry and metabolomics 69% · Simulation 75% · Tabular 65%
Provenance · 1 source records, 19 field assertions
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