← Search

Table · dataset · 2026

Supplementary material: A trial emulation study indirectly comparing tafamidis and acoramidis in transthyretin amyloid cardiomyopathy: the ReplicATTR study

Listed in figshare

Description

<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.

Read the rest (2 more)

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>

Links

Where it is published

Catalogue records · 1

Topics

Inferred from text
Cardiomyopathy 75% · Medical biochemistry and metabolomics 69% · Simulation 75% · Tabular 65%
Provenance · 1 source records, 19 field assertions
SourceKeyLast seenRaw
figshareoai:figshare.com:article/3372652911 d agoJSON v1
FieldAssertionExtractorEvidence
concepts[disease].local:disease:cardiomyopathyenrichment · figshare comkeyword-concept-rules@1.0.0title+description (75%)
concepts[field].anzsrc:group:3205enrichment · figshare comtaxonomy-embedding@1.0.0title+keywords+description (69%)
concepts[field].local:field:astronomymapping · figshare comconnector:figshare_com@1.0.0
concepts[field].local:field:chemistrymapping · figshare comconnector:figshare_com@1.0.0
concepts[field].local:field:computer-science-aimapping · figshare comconnector:figshare_com@1.0.0
concepts[field].local:field:earth-environmentalmapping · figshare comconnector:figshare_com@1.0.0
concepts[field].local:field:economics-financemapping · figshare comconnector:figshare_com@1.0.0
concepts[field].local:field:engineeringmapping · figshare comconnector:figshare_com@1.0.0
concepts[field].local:field:humanitiesmapping · figshare comconnector:figshare_com@1.0.0
concepts[field].local:field:life-sciencesmapping · figshare comconnector:figshare_com@1.0.0
concepts[field].local:field:medicine-healthmapping · figshare comconnector:figshare_com@1.0.0
concepts[field].local:field:ocean-atmosphericmapping · figshare comconnector:figshare_com@1.0.0
concepts[field].local:field:social-sciencemapping · figshare comconnector:figshare_com@1.0.0
concepts[method].local:method:simulationenrichment · figshare comkeyword-concept-rules@1.0.0title+description (75%)
concepts[modality].local:modality:tabularenrichment · figshare comkeyword-concept-rules@1.0.0title+description (65%)
descriptionsource · figshare comconnector:figshare_com@1.0.0/metadata/dc/description
license_textsource · figshare comconnector:figshare_com@1.0.0
publication_datesource · figshare comconnector:figshare_com@1.0.0
titlesource · figshare comconnector:figshare_com@1.0.0/metadata/dc/title