What is the role of axicabtagene ciloleucel (Yescarta) in chimeric antigen receptor (CAR) T-cell therapy?

Updated: Dec 17, 2020
  • Author: Sameh Gaballa, MD, MS; Chief Editor: Emmanuel C Besa, MD  more...
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The second CAR T-cell therapy to enter clinical practice, axicabtagene ciloleucel (Yescarta), was approved by the FDA in October 2017. Axicabtagene ciloleucel is CD19-directed with a CD28 co-stimulatory molecule and is indicated for use in adults with DLBCL who have not responded to or who have relapsed after at least two other lines of therapy. Approved uses include DLBCL, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma. Axicabtagene ciloleucel is not indicated for the treatment of primary central nervous system lymphoma. [13]

The ZUMA-1 trial in refractory aggressive non-Hodgkin lymphoma (DLBCL, primary mediastinal B-cell lymphoma, or transformed follicular lymphoma) enrolled 111 patients, 101 of whom received axicabtagene ciloleucel. The objective response rate (ORR)—consistent across disease subtypes and other key covariates—was 82%. [14] At a median follow up of 27.1 months, 83% of patients had a response and 58% had a complete response. The median duration of response was 11.1 months and the median overall survival was not reached. [15]

Since the approval of axicabtagene ciloleucel, data in patients treated outside of clinical trials have become available. These data show that patients treated in the “real world” have outcomes similar to those reported in the ZUMA-1 trial, with approximately 40-50% of patients remaining in remission at 12 months. Patients with a baseline high lactate dehydrogenase level or poor performance status were found to have the highest risk of progression or death after CAR T-cell therapy. [16, 17]

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