San Diego—A follow-up analysis of the ZUMA-1 trial reported 51% of patients receiving axicabtagene ciloleucel, an autologous anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, were still alive 2 years after treatment.

The updated findings, with a median follow-up of 27.1 months, were presented at the 2018 ASH Annual Meeting by lead investigator Sattva S. Neelapu, MD, Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, Houston, Texas, and published in The Lancet Oncology (2018 Dec 2. Epub ahead of print).

The ZUMA-1 Trial

Patients aged 18 or older with histologically confirmed large B-cell lymphoma, including diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma, and transformed follicular lymphoma, were eligible for the ZUMA-1 trial. A total of 119 patients were enrolled between May 19, 2015, and Sept 15, 2016 and 108 received axicabtagene ciloleucel across phases 1 and 2.

The primary endpoints of the trial were safety for phase 1 and the proportion of patients achieving an objective response for phase 2. The key secondary endpoints were overall survival, progression-free survival, and duration of response.

Long-Term Durable Responses and Manageable Safety Profile

At a median follow-up of 27.1 months, 101 patients were assessable for activity in phase 2. Of these patients, 84 (83%) had an objective response, and 59 (58%) had a complete response. These results were similar to previous findings at a follow-up of 15.4 months, at which time the objective response rate was 82% and complete response rate was 58%.

The updated analysis reported the median duration of response was 11.1 months. Median overall survival was not reached, and the median progression-free survival was 5.9 months.

A total of 52 of 108 patients assessable for safety in phases 1 and 2 had grade 3 or worse serious adverse events. Grade 3 or worse cytokine release syndrome occurred in 12 (11%) patients, and grade 3 or worse neurological events in 35 (32%) patients.

Since the previous analysis, additional serious adverse events were reported in 4 patients, none of which were found to be treatment-related. Two treatment-related deaths were previously reported in the 1-year analysis, but no new treatment-related deaths occurred during the additional follow-up.

“These 2-year follow-up data from ZUMA-1 suggest that axicabtagene ciloleucel can induce durable responses and a median overall survival of greater than 2 years, and has a manageable long-term safety profile in patients with relapsed or refractory large B-cell lymphoma,” Dr Neelapu and colleagues concluded.—Janelle Bradley

Neelapu SS, Ghobadi A, Jacobson C, et al. 2-Year Follow-up and High-Risk Subset Analysis of Zuma-1, the Pivotal Study of Axicabtagene Ciloleucel (Axi-Cel) in Patients with Refractory Large B Cell Lymphoma. Presented at: the 60th ASH Annual Meeting and Exposition; December 1-4, 2018; San Diego, CA. Abstract 2967.