Early results of the first report on the chimeric antigen receptor (CAR) T-cell therapy axicabtagene ciloleucel (axi-cel) to treat relapsed or refractory (R/R) follicular lymphoma (FL) in the real-world setting demonstrated effectiveness and safety profiles consistent with those observed in the ZUMA-5 trial, according to research at the ASCO Annual Meeting 2023.

“Approval [of axi-cel] was supported by the primary analysis of the pivotal ZUMA-5 trial, where in 94% of patients receiving axi-cel achieved an overall response, with 76% achieving a complete response,” explained Caron Alyce Jacobson, MD, MMSc, medical director of the immune effector cell therapy program and senior physician with Dana-Farber Cancer Institute in Boston, MA. “A postauthorization safety study of axi-cel is currently ongoing, using the Center for International Blood and Marrow Transplant Research, or CIBMTR, to collect long-term safety and effectiveness information postinfusion for up to 15 years.”

A total of 151 patients receiving axi-cel for R/R FL in the real-world setting between March 2021 and October 2022 were identified from the CIBMTR registry and included in the analysis.

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Patients had a median age of 62 years. Most (98%) had an ECOG performance score of 0-1. Elevated lactate dehydrogenase and chemo-resistance were observed in 33% and 66% of patients, respectively, and clinically significant comorbidities were reported in 75% of patients. Among those included in the analysis, 40% of patients would have been considered ineligible for ZUMA-5, mainly due to comorbidities.

The researchers found overall response rates (ORR) and complete response (CR) rates (ORR, 93%; 95% CI, 88-97; CR, 84%; 95% CI, 77-89) were comparable to those of the ZUMA-5 trial, regardless of ZUMA-5 eligibility or age.

They also demonstrated that real-world time-to-event outcomes, including duration of response, progression-free survival, and overall survival (medians not reached), were comparable to those of the ZUMA-5 trial, including in patients age ≥65 years and those who would have been ineligible. The median follow-up duration was 6.2 months.

Researchers additionally reported that real-world safety outcomes with axi-cel in these patients were comparable with those observed in the ZUMA-5 trial, including grade ≥3 cytokine release syndrome (2%), grade ≥3 immune effector cell-associated neurotoxicity syndrome (12%) , and incidences of prolonged cytopenias (neutropenia, 4%; thrombocytopenia, 9%).

“In summary, 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,” concluded the presenter.

Disclosure: This research was supported by Kite, a Gilead company. Please see the original reference for a full list of disclosures.


Jacobson CA, Hemmer MT, Hu Zh, et al. Real-world early outcomes of axicabtagene ciloleucel for relapsed or refractory (R/R) follicular lymphoma (FL). ASCO 2023. June 2-6, 2023. Abstract 7509.