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Axi-Cel Highly Effective as First-line Option in High-risk Large B-Cell Lymphoma


Research on axicabtagene ciloleucel (axi-cel) shows its effectiveness as a first-line option in patients with high-risk large B-cell lymphoma (LBCL).

The phase 2 findings confirm research that was presented in December at the 63rd Annual American Society of Hematology Meeting and Exposition. The study was published Monday in Nature Medicine.

Sixty percent of patients with LBCL have good responses to standard first-line chemoimmunotherapy regimens, but outcomes are lower for those with high-risk LBCL, a subgroup of the disease. These patients have double- or triple-hit lymphoma (meaning gene arrangements of MYC and BCL2 and/or BCL6) or additional clinical risk factors identified by the International Prognostic Index (IPI) or interim positron emission tomography scan.

Historically, less than half of these patients achieve long-term disease remission with standard approaches.

Axi-cel, an autologous anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, is already approved for patients with relapsed/refractory LBCL after 2 or more systemic lines of therapy.

In this multicenter single-arm ZUMA-12 study (NCT03761056), 40 patients with high-risk LBCL were enrolled and treated with axi-cel. Ninety-five percent had stage III/IV disease, 25% had double or triple-hit status per central assessment, and 78% had an IPI score of at least 3.

Results for the 40 patients treated with axi-cel showed:

  • 89% had an objective response
  • 78% experienced a complete response
  • 73% had an ongoing response at data cutoff, after a median follow-up of 15.9 months

The estimated overall survival rate at 12 months was 91%, and although the median for duration of response, event-free survival, and progression-free survival were not reached, 12-month estimates were 81%, 73%, and 75%, respectively.

The treatment was well tolerated with no new safety signals and no treatment-related grade 5 adverse events. Grade ≥3 cytokine release syndrome and neurologic events occurred in 3 patients (8%) and 9 patients (23%), respectively.

Robust CAR T-cell expansion occurred in all patients, with a median time to peak of 8 days.

“Existing treatments for LBCL consist of 6 months of chemotherapy,” Sattva S. Neelapu, MD, professor and deputy department chair in the Department of Lymphoma/Myeloma in the Division of Cancer Medicine at the University of Texas MD Anderson Cancer Center, said in a statement. “These results provide evidence that axi-cel may offer effective responses in one treatment and eliminate the need for patients to be exposed to other therapies."

He noted that randomized clinical trials are still necessary to confirm the findings.


Neeolapu SS, Dickinson M, Munoz J, et al. Axicabtagene ciloleucel as first-line therapy in high-risk large B-cell lymphoma: the phase 2 ZUMA-12 trial. Nat Med. Published online March 21, 2022. doi:10.1038/s41591-022-01731-4

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