Five-year follow-up of ZUMA-1 supports the curative potential of axicabtagene ciloleucel in refractory large B-cell lymphoma

Sattva S. Neelapu, Caron A. Jacobson, Armin Ghobadi, David B. Miklos, Lazaros J. Lekakis, Olalekan O. Oluwole, Yi Lin, Ira Braunschweig, Brian T. Hill, John M. Timmerman, Abhinav Deol, Patrick M. Reagan, Patrick Stiff, Ian W. Flinn, Umar Farooq, Andre H. Goy, Peter A. McSweeney, Javier Munoz, Tanya Siddiqi, Julio C. ChavezAlex F. Herrera, Nancy L. Bartlett, Adrian A. Bot, Rhine R. Shen, Jinghui Dong, Kanwarjit Singh, Harry Miao, Jenny J. Kim, Yan Zheng, Frederick L. Locke

Research output: Contribution to journalArticlepeer-review

33 Scopus citations


In phase 2 of ZUMA-1, a single-arm, multicenter, registrational trial, axicabtagene ciloleucel (axi-cel) autologous anti-CD19 chimeric antigen receptor (CAR) T-cell therapy demonstrated durable responses at 2 years in patients with refractory large B-cell lymphoma (LBCL). Here, we assessed outcomes in ZUMA-1 after 5 years of follow-up. Eligible adults received lymphodepleting chemotherapy followed by axi-cel (2 × 106 cells per kg). Investigator-assessed response, survival, safety, and pharmacokinetics were assessed in patients who had received treatment. The objective response rate in these 101 patients was 83% (58% complete response rate); with a median follow-up of 63.1 months, responses were ongoing in 31% of patients at data cutoff. Median overall survival (OS) was 25.8 months, and the estimated 5-year OS rate was 42.6%. Disease-specific survival (excluding deaths unrelated to disease progression) estimated at 5 years was 51.0%. No new serious adverse events or deaths related to axi-cel were observed after additional follow-up. Peripheral blood B cells were detectable in all evaluable patients at 3 years with polyclonal B-cell recovery in 91% of patients. Ongoing responses at 60 months were associated with early CAR T-cell expansion. In conclusion, this 5-year follow-up analysis of ZUMA-1 demonstrates sustained overall and disease-specific survival, with no new safety signals in patients with refractory LBCL. Protracted B-cell aplasia was not required for durable responses. These findings support the curative potential of axi-cel in a subset of patients with aggressive B-cell lymphomas. This trial was registered at, as #NCT02348216.

Original languageEnglish
Pages (from-to)2307-2315
Number of pages9
Issue number19
StatePublished - May 11 2023


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