Axicabtagene ciloleucel in the non-trial setting: Outcomes and correlates of response, resistance, and toxicity

Caron A. Jacobson, Bradley D. Hunter, Robert Redd, Scott J. Rodig, Pei Hsuan Chen, Kyle Wright, Mikel Lipschitz, Jerome Ritz, Yusuke Kamihara, Philippe Armand, Sarah Nikiforow, Michael Rogalski, Joseph Maakaron, Samantha Jaglowski, Marcela V. Maus, Yi Bin Chen, Jeremy S. Abramson, Justin Kline, Elizabeth Budde, Alex HerreraMatthew Mei, Jonathon B. Cohen, Stephen D. Smith, David G. Maloney, Ajay K. Gopal, Matthew J. Frigault, Utkarsh H. Acharya

Research output: Contribution to journalArticlepeer-review

215 Scopus citations


PURPOSE: Axicabtagene ciloleucel (axi-cel) was approved by the Food and Drug Administration for relapsed aggressive B-cell non-Hodgkin lymphoma in part on the basis of durable remission rates of approximately 40% in a clinical trial population. Whether this efficacy, and the rates of toxicity, would be consistent in a postcommercial setting, with relaxed eligibility criteria and bridging therapy, is unknown. This study describes the efficacy and safety correlates and outcomes in this setting.

PATIENTS AND METHODS: One hundred twenty-two patients from 7 medical centers in the United States were treated with axi-cel and were included in a modified intent-to-treat (mITT) analysis. Seventy-six patients (62%) were ineligible for the ZUMA-1 trial. Response and toxicity rates, duration of response (DOR), survival, and covariates are described on the basis of the mITT population. Correlative studies on blood and tumor samples were performed to investigate potential biomarkers of response and resistance.

RESULTS: Median follow-up was 10.4 months. In the mITT population, the best overall and complete response (CR) rates were 70% and 50%, respectively. Median DOR and progression-free survival (PFS) were 11.0 and 4.5 months in all patients and were not reached (NR) in CR patients. Median overall survival (OS) was NR; 1-year OS was 67% (95% CI, 59% to 77%). Although response rates were similar in the ZUMA-1-eligible and ZUMA-1-ineligible groups (70% v 68%), there was a statistically significant improvement in CR rate (63% v 42%, P = .016), DOR (median, NR v 5.0 months; P = .014), PFS (median, NR v 3.3 months; P = .020), and OS (1-year OS, 89% v 54%; P < .001) in patients who were ZUMA-1 eligible. Rates of grade ≥ 3 cytokine release syndrome and neurotoxicty were 16% and 35%, respectively.

CONCLUSION: Axi-cel yields similar rates of overall response and toxicity in commercial and trial settings, although CR rates and DOR were more favorable in patients eligible for ZUMA-1.

Original languageEnglish (US)
Pages (from-to)3095-3106
Number of pages12
JournalJournal of Clinical Oncology
Issue number27
StatePublished - Sep 20 2020

Bibliographical note

Funding Information:
Supported in part by funding from the Cancer Clinical Investigator Team Leadership Award from the National Cancer Institute though a supplement to P30CA006516.

Publisher Copyright:
Copyright © 2020 American Society of Clinical Oncology. All rights reserved.


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