Unravelling real-world safety and effectiveness of axi-cel in LBCL

A large real-world study demonstrated that the safety and effectiveness of axicabtagene ciloleucel (axi-cel) in patients with relapsed or refractory large B-cell lymphoma (LBCL) was comparable with the outcomes of the ZUMA-7 trial.

In many countries, the anti-CD19 CAR T-cell therapy axi-cel has been approved for the second-line treatment of patients with relapsed or refractory LBCL, based on the results of the ZUMA-7 trial [1]. Dr Dasom Lee (Stanford University, CA, USA) and colleagues studied the real-world effectiveness and safety of axi-cel as a second-line treatment option for adult patients with LBCL (n=461) [2]. “About half of the patients we included would have been ineligible for ZUMA-7,” emphasised Dr Lee. Thus, this real-world study represents a broader range of patients.

At 6 months, the cumulative incidence of time to overall response and complete response rates were 79% and 61%, respectively. In addition, the 1-year duration of response rate was 66%, while the 1-year event-free survival and overall survival (OS) rates were 54% and 74%, respectively. “We observed significantly shorter OS in patients with cardiac comorbidities (HR 1.70; 95% CI 1.03–2.80), whereas hepatic, pulmonary, or infectious comorbidities did not seem to influence effectiveness outcomes,” mentioned Dr Lee.

Cytokine release syndrome (CRS) was observed in 88% of patients, with 5% grade ≥3 events, and immune effector cell-associated neurotoxicity syndrome (ICANS) was documented in 48% of patients, with 23% grade ≥3 events. Other adverse events of interest were prolonged neutropenia (6%), prolonged thrombocytopenia (11%), and second primary malignancies (3%). “Patients above 65 years of age appeared to have an increased risk of ICANS (OR 2.25; 95% CI 1.51–3.36), and patients with infections were more likely to have prolonged neutropenia (HR 4.38; 95% CI 1.13–17.00),” said Dr Lee.

In conclusion, this large real-world study confirmed the effectiveness and safety of axi-cel as second-line therapy for patients with LBCL and highlighted some associations between the treatment and patient characteristics. “We did not have many patients with renal disease or with ECOG performance status ≥2,” mentioned Dr Lee. “Future studies need to investigate the real-world value of axi-cel in these specific patient populations.”

1. Westin JR, et al. N Engl J Med 2023;389:148-157.
2. Lee D, et al. Real-world effectiveness and safety outcomes among key subgroups of second-line axicabtagene ciloleucel (axi-cel) for patients with relapsed/refractory (R/R) large B-cell lymphoma. S237, EHA2025 Congress, 12–15 June, Milan, Italy.

Medical writing support was provided by Robert van den Heuvel.
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Posted on 18 June 202518 June 2025