Axi-Cel Shows Favorable Safety, Efficacy in Real-World Study of Early FL Outcomes


The autologous CAR T-cell therapy axicabtagene ciloleucel (axi-cel) demonstrated effectiveness and safety consistent with the ZUMA-5 trial when used in real-world settings to treat a broader population of patients with follicular lymphoma (FL).

In the largest report of real-world axicabtagene ciloleucel (axi-cel) outcomes among patients with relapsed or refractory follicular lymphoma (R/R FL), including patients who would have been ineligible for the pivotal ZUMA-5 trial, the chimeric antigen receptor (CAR) T-cell therapy showed safety and efficacy consistent with trial outcomes.1 The findings were presented during a poster discussion session at the 2023 American Society of Clinical Oncology Annual Meeting.

In the pivotal ZUMA-5 trial of axi-cel in R/R FL following at least 2 lines of therapy, the primary analysis showed a 94% objective response rate (ORR) and a 79% complete response (CR) rate in patients treated with axi-cel.2 Regarding safety, cytokine release syndrome (CRS) grade 3 or higher occurred in 6% of patients, and neurologic events occurred in 15% of patients.

In the new analysis, the patient population is significantly broader than that of the ZUMA-5 trial. Of 230 patients in the real-world study population, 40% would have been excluded from the ZUMA-5 trial, mainly due to comorbidities.

The median patient age was 62 years, and 60% of patients in the study were male. Patients had a median of 4 lines of therapy prior to receiving axi-cel, 14% had undergone prior autologous stem cell transplantation, and 9% received bridging therapy. Patients who received previous nontransplant cellular therapy, including prior CAR T-cell therapy, were excluded.

Among 151 patients with follow-up data at a median of 6.2 months, the ORR was 93% (95% CI, 88% to 97%) and the CR rate was 84% (95% CI, 77% to 89%). The estimated progression-free survival (PFS) and overall survival (OS) were 88% (95% CI, 81% to 92%) and 96% (95% CI, 91% to 98%).

“Despite a broader patient population and relatively limited follow-up in the real world, early results demonstrate favorable effectiveness and safety outcomes with axi-cel in patients with relapsed or refractory follicular lymphoma that are consistent with those observed in the pivotal ZUMA-5 trial, supporting postauthorization use of this CAR T-cell therapy in this setting,” said lead study author and presenter Caron A. Jacobson, MD, MMSc, medical director of the Immune Effector Cell Therapy Program at Dana-Farber Cancer Institute and assistant professor of medicine at Harvard Medical School.

CRS considered grade 3 or higher by ASTCT consensus occurred in 2% of patients (95% CI, 0% to 6%), and immune effector cell-associated neurotoxicity syndrome (ICANS) occurred in 13% of patients (95% CI, 8% to 19%). The median cumulative incidence estimates of CRS and ICAN resolution were 5 days and 4 days, respectively. Among 150 patients who were alive at day 30, prolonged cytopenia occurred in 11%.

Notably, PFS and OS at 6 months post-treatment were similar regardless of patient eligibility by ZUMA-5 trial criteria. However, those who would have been eligible for ZUMA-5 showed lower rates of grade 3 or higher ICANS and quicker ICANS resolution.


1. Jacobsen CA, Hemmer MT, Hu ZH, et al. Real-world early outcomes of axicabtagene ciloleucel for relapsed or refractory (R/R) follicular lymphoma (FL). J Clin Oncol. 2023;41(suppl 16):7509. doi:10.1200/JCO.2023.41.16_suppl.7509

2. Jacobsen CA, Chavez JC, Sehgal AR, et al. Axicabtagene ciloleucel in relapsed or refractory indolent non-Hodgkin lymphoma (ZUMA-5): a single-arm, multicentre, phase 2 trial. Lancet Oncol. 2022;23(1):91-103. doi:10.1016/S1470-2045(21)00591-X

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