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Abstract Efficacy of Axicabtagene ciloleucel (Axi-cel) as a frontline regimen for high-risk large B-cell lymphoma (LBCL) has been described in ZUMA-12 single-arm trial, yet there is a paucity of data on head-to-head effectiveness comparison between axi-cel vs. conventional therapy. We conducted an external comparator arm (ECA) study to compare overall survival (OS) and progression-free survival (PFS) in patients treated with axi-cel from ZUMA-12 with ECA treated with conventional therapies from SMC-LCS (Samsung Medical Center-Lymphoma Cohort Study 2017–2023); published summary data from ZUMA-12 and individual patient data from SMC-LCS were used. Matching-adjusted indirect comparison weighting was used to compare OS and PFS, adjusting for the baseline characteristics. Of 279 patients with high-risk LBCL in SMC-LCS, 45 fulfilled ZUMA-12 eligibility criteria. Mortality rates were 13.5% in ZUMA-12 and 49.5% in ECA, corresponding to a lower hazard of death for axi-cel (hazard ratio 0.30 95% CI 0.13–0.73). Median PFS of ZUMA-12 was not reached vs 2.7 months in ECA, corresponding to improved PFS for axi-cel (hazard ratio 0.23 0.11–0.46). This study leveraged real-world data as a source for external comparator to present clinically meaningful evidence on the comparative effectiveness of axi-cel for high-risk LBCL.
Shin et al. (Mon,) studied this question.