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Chimeric antigen receptor T‐cell (CAR‐T) therapy is becoming an effective technique for the treatment of patients with relapsed/refractory hematologic malignancies. After analyzing patients with tumor progression and sustained remission after CAR‐T cell therapy, many factors were found to be associated with the efficacy of CAR‐T therapy. This paper reviews the factors affecting the effect of CAR‐T such as tumor characteristics, tumor microenvironment and immune function of patients, CAR‐T cell structure, construction method and in vivo expansion values, lymphodepletion chemotherapy, and previous treatment, and provides a preliminary outlook on the corresponding therapeutic strategies. [ABSTRACT FROM AUTHOR]

Aim: Cost–effectiveness analysis (CEA) was performed to compare axicabtagene ciloleucel (axi-cel) with tisagenlecleucel (tisa-cel) and lisocabtagene (liso-cel) for treatment of relapsed or refractory large B-cell lymphoma in adult patients after ≥2 lines of therapy in Japan. Materials & methods: Cost–effectiveness analysis was conducted using the partition survival mixture cure model based on the ZUMA-1 trial and adjusted to the JULIET and TRANSCEND trials using matching-adjusted indirect comparisons. Results & conclusion: Axi-cel was associated with greater incremental life years (3.13 and 2.85) and incremental quality-adjusted life-years (2.65 and 2.24), thus generated lower incremental direct medical costs (-$976.29 [-¥137,657] and -$242.00 [-¥34,122]), compared with tisa-cel and liso-cel. Axi-cel was cost-effective option compared with tisa-cel and liso-cel from a Japanese payer's perspective. This is the first study comparing cost–effectiveness among different CD19-targeted chimeric antigen receptor T-cell therapies in Japan. Axi-cel was associated with additional life years (LYs) and quality-adjusted life years (QALYs) gained and decreased costs compared with either tisa-cel or liso-cel, demonstrating that it is a cost-effective treatment option among currently available chimeric antigen receptor T-cell therapies in Japan which may improve life expectancy. In the base case, axicabtagene ciloleucel (axi-cel) was found to be highly cost-effective versus tisagenlecleucel (tisa-cel) and lisocabtagene (liso-cel). Axi-cel was associated with more LYs (3.13 and 2.85) and QALYs (2.65 and 2.24), and lower direct medical costs (-$976.29 [-¥137,657] and -$242.00 [-¥34,122]), compared with tisa-cel and liso-cel, respectively. The gain in discounted LYs and QALYs was predominantly attributable to pre-progression survival status for axi-cel. Compared with tisa-cel and liso-cel, patients treated with axi-cel experienced a longer progression-free state and improved QALYs (discounted LYs: 3.36 and 2.66; QALYs 2.75 and 2.17, respectively). The analyses showed that patients who received axi-cel incurred lower total discounted costs by -$976.29 (-¥137,657) and -$242.00 (-¥34,122) versus tisa-cel and liso-cel ($280,875.73 vs $281,852.02 and $280,673.52 vs $280,915.52), respectively. These reductions in total discounted costs for axi-cel were driven by apheresis costs and end-of-life care costs versus tisa-cel, and by end-of-life care costs versus liso-cel, respectively. [ABSTRACT FROM AUTHOR]

Abstract Chimeric antigen receptor T‐cell (CAR‐T) therapy is becoming an effective technique for the treatment of patients with relapsed/refractory hematologic malignancies. After analyzing patients with tumor progression and sustained remission after CAR‐T cell therapy, many factors were found to be associated with the efficacy of CAR‐T therapy. This paper reviews the factors affecting the effect of CAR‐T such as tumor characteristics, tumor microenvironment and immune function of patients, CAR‐T cell structure, construction method and in vivo expansion values, lymphodepletion chemotherapy, and previous treatment, and provides a preliminary outlook on the corresponding therapeutic strategies.