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Anti-CD19 CAR T-cell therapy for patients with primary or secondary CNS lymphoma

May 3, 2024
Learning objective: After reading this article, learners will be able to cite a new development in the treatment of lymphoma.

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Patients with relapsed/refractory (R/R) primary central nervous system lymphoma (PCNSL) or secondary central nervous system lymphoma (SCNSL) have a dismal prognosis; as such, there is a need for novel treatment strategies for these patients.1 Several trials, including  ZUMA-1 (NCT02348216) and  JULIET (NCT02445248), have demonstrated the efficacy of anti-CD19 chimeric antigen receptor (CAR) T-cell therapy in patients with relapsed/refractory large B-cell lymphoma1; however, there is a lack of data on the safety and efficacy of anti-CD19 CAR T-cell therapy in patients with PCNSL/SCNSL.1

During the 50th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), Ossami Saidy presented results from an analysis of CD19-targeted CAR T-cell therapy in patients with PCNSL or SCNSL from the EBMT and GoCART coalition.1 We summarize this presentation below.

Study design1

  • This analysis included adult patients aged ≥18 years with PCNSL or SCNSL who received their first CAR T-cell therapy between January 2018 and February 2023.
  • The primary endpoints were overall survival and progression-free survival.
  • Secondary endpoints included relapse incidence, non-relapse morality, and toxicity.
  • Data was obtained from the EBMT registry and a questionnaire.

Key findings1

Patient characteristics

A total of 95 patients with a median age of 62.6 years were included in this analysis. (Table 1).

Table 1. Patient characteristics*

Patient characteristics, % (unless otherwise specified)

All patients

(N = 95)

Median age, years (range)

62.6 (23–80)

              Aged >70 years




              Male, n


              Female, n


Type of CNS lymphoma






ECOG Performance Status ≥2


Number of prior lines of therapy








Previously auto-HSCT§


Remission status at CAR T-cell infusion


              Stable disease/progressive disease


              Partial remission


              Complete remission


Auto-HSCT, autologous hematopoietic stem cell transplantation; CAR, chimeric antigen receptor; CNS, central nervous system; ECOG, Eastern Cooperative Oncology Group; PCNSL, primary central nervous system lymphoma; SCNSL, secondary CNSL.

*Adapted from Ossami Saidy.1
n = 93.
n = 69.
§n = 93.
n = 89.

Survival outcomes

  • After a median follow-up of 20.1 months, the 2-year progression-free survival and overall survival rates were 38.1% and 48.4%, respectively (Figure 1).
  • Patients who received CAR T-cell therapy in complete or partial remission had improved survival outcomes compared with patients who had stable or progressive disease (Figure 1).

Figure 1. 2-year survival rates in patients with PCNSL or SCNSL* 

CR, complete remission; NRM, non-relapse mortality; OS, overall survival; PCNSL, primary central nervous system lymphoma; PD, progressive disease; PFS, progression-free survival; PR, partial remission; RI, relapse incidence; SCNSL, secondary central nervous system lymphoma; SD, stable disease.

*Data from Ossami Saidy1


After a median follow-up of 27 months, the incidence rates of immune effector cell-associated neurotoxicity syndrome at 3 and 15 days were 17% and 43%, respectively.

Key learnings
  • Results suggest that anti-CD19 CAR T-cell therapy is effective in patients with PCNSL or SCNSL, with similar outcomes to patients with R/R large B-cell lymphoma without CNS involvement.
  • Anti-CD19 CAR T-cell therapy should therefore be considered in all patients with R/R LBCL and CNS involvement.  

  1. Ossami Saidy A. Efficacy and toxicity of CAR T-cell therapy in patients with primary and secondary CNS lymphoma. Oral abstract #GCC1-4. 50th Annual Meeting of the European Society for Blood and Marrow Transplantation. Apr 17, 2024. Glasgow, UK.

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