2026-10-14T06:42:54Z https://api.figshare.com/v2/oai

oai:figshare.com:article/33726529 2026-09-14T09:47:23Z category_24463 portal_1083 item_type_3 month_year_09_2026

Supplementary material: A trial emulation study indirectly comparing tafamidis and acoramidis in transthyretin amyloid cardiomyopathy: the ReplicATTR study Philippe Debonnaire (24873848) Peter-Paul Zwetsloot (10056334) Roman Pfister (24873857) Pau Llacer (24873862) Alberto Cipriani (24873864) Paolo Milani (24873865) Diana Bonderman (24873873) Christoph Ohlmeier (24873877) Thomas Evers (24873880) Richard Wyss (24873882) Elisabetta Patorno (24873885) Riddhi Doshi (20357427) Julien Beisel (16496523) Craig I Coleman (24873896) Carlos Aguiar (24873907) Tomas Ripoll-Vera (24873916) Cardiology (incl. cardiovascular diseases) acoramidis all-cause mortality ATTR-CM comparative effectiveness research tafamidis target trial emulation transthyretin amyloid cardiomyopathy transthyretin stabilizer These are peer-reviewed supplementary materials for the article 'A trial emulation study indirectly comparing tafamidis and acoramidis in transthyretin amyloid cardiomyopathy: the ReplicATTR study' published in the Journal of Comparative Effectiveness Research.<ul><li>Supplemental figure 1: Pictorial representation of the trial emulation study comparing tafamidis and acoramidis in transthyretin amyloid cardiomyopathy using calibration and indirect treatment comparison methods</li><li>Supplemental table 1: Exclusion criteria to align the real-world tafamidis cohort with ATTR-ACT</li><li>Supplemental table 2: Exclusion criteria to align the real-world tafamidis cohort with ATTRibute-CM</li><li>Supplemental table 3: Attrition for the real-world tafamidis cohort using ATTR-ACT criteria</li><li>Supplemental table 4: Attrition for the real-world tafamidis cohort using ATTRibute-CM criteria</li><li>Supplemental table 5: Distributions used for the Monte Carlo simulation</li><li>Supplemental table 6: Pre- and post-ATTR-ACT trial-emulated real-world cohort characteristics compared to ATTR-ACT trial aggregate data</li><li>Supplemental table 7: Pre- and post-ATTRibute-CM trial-emulated real-world cohort characteristics compared to ATTRibute-CM trial aggregate data</li><li>Supplemental table 8: 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>References</li></ul>Aim: 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). Materials & methods: 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. 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. Results: 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. Conclusion: 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. 2026-09-14T09:47:23Z Dataset Dataset 10.6084/m9.figshare.33726529.v1 https://figshare.com/articles/dataset/Supplementary_material_A_trial_emulation_study_indirectly_comparing_tafamidis_and_acoramidis_in_transthyretin_amyloid_cardiomyopathy_the_ReplicATTR_study/33726529 CC BY-NC-ND 4.0