Axicabtagene ciloleucel (axi-cel), an autologous anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, appears to have curative potential for a subset of patients with refractory large B-cell lymphoma (LBCL), according to research published in Blood.

On the basis of data from ZUMA-1 ( Identifier: NCT02348216), an open-label phase 1/2 study of axi-cel in patients with refractory non-Hodgkin lymphoma, axi-cel was approved for some patients who had received at least 2 prior systemic therapy lines.

However, it was previously unclear whether axi-cel induced long-term responses among treated patients. For this 5-year analysis of ZUMA-1, researchers aimed to determine the long-term safety and efficacy of axi-cel among patients with LBCL.

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Overall, 101 patients were enrolled and treated on ZUMA-1. At baseline, the median age was 58 years (range, 23-76), 67% of patients were male sex, 76% of patients had diffuse LBCL, 89% of patients had received prior platinum-based therapy, and 8% of patients had lymphoma present in the bone marrow.

Although the objective response rate was 83% on initial analysis, after a median follow-up of 63.1 months, responses were ongoing in 31% of patients. The median overall survival in the cohort was 25.8 months, with an estimated 5-year overall survival rate of 42.6%. The 5-year disease-specific survival rate was estimated to be 51%.

Further analysis showed that early CAR T-cell expansion was linked with an ongoing response at 60 months. The authors also noted that there were no new safety signals during the 5-year follow up.

“Importantly,” the authors wrote, “long-term results from the ZUMA-1 trial support the curative potential of axi-cel for a large proportion of patients with aggressive lymphomas.”
Disclosures: This research was supported by Kite, a Gilead Company. Please see the original reference for a full list of disclosures.


Neelapu SS, Jacobson CA, Ghobadi A, et al. Five-year follow-up of ZUMA-1 supports the curative potential of axicabtagene ciloleucel in refractory large B-cell lymphoma. Blood. 2023;141(19):2307-2315. doi:10.1182/blood.2022018893