Axicabtagene Ciloleucel in Patients Ineligible for ZUMA-1 Because of CNS Involvement and/or HIV: A Multicenter

Carlen A Yuen1, Jing-Mei Hsu2, Koen Van Besien2

  • 1Department of Neurology and Herbert Irving Comprehensive Cancer Center, Columbia University Vagelos College of Physicians and Surgeons.

Insights

Chimeric antigen receptor T-cell therapy shows promise for treating secondary central nervous system lymphoma (SCNSL). This approach yielded significant response rates in SCNSL patients, including those with human immunodeficiency virus, suggesting its potential as a life-saving treatment.

Area of Science:

  • Oncology
  • Immunotherapy
  • Hematology

Background:

  • Secondary central nervous system lymphoma (SCNSL) presents a significant clinical challenge with a poor prognosis.
  • Existing therapeutic options for SCNSL are limited, necessitating the exploration of novel treatment strategies.
  • The pivotal ZUMA-1 trial, leading to FDA approval of axicabtagene ciloleucel, excluded patients with SCNSL and human immunodeficiency virus (HIV).

Purpose of the Study:

  • To evaluate the efficacy and safety of axicabtagene ciloleucel in patients with SCNSL.
  • To assess the outcomes of chimeric antigen receptor T-cell therapy in a cohort of SCNSL patients, including those with HIV co-infection.

Main Methods:

  • A multi-institutional retrospective study was conducted.
  • Fourteen patients diagnosed with SCNSL were treated with axicabtagene ciloleucel.
  • Outcomes, including rates of severe neurotoxicity and complete response, were analyzed.

Main Results:

  • The study observed a severe neurotoxicity rate of 32% and a complete response rate of 58% in SCNSL patients treated with axicabtagene ciloleucel.
  • Three patients in the cohort had human immunodeficiency virus (HIV) and were included in the analysis.
  • These response and toxicity rates were comparable to those reported in the ZUMA-1 trial at a median follow-up of 5.9 months.

Conclusions:

  • Chimeric antigen receptor T-cell therapy, specifically axicabtagene ciloleucel, demonstrates potential as a life-saving treatment for patients with SCNSL.
  • The therapy should not be withheld from SCNSL patients, even those with HIV co-infection, given the promising outcomes.
  • Further prospective studies are warranted to confirm these findings in larger cohorts.