Efficacy and safety of tisagenlecleucel in Japanese pediatric and young adult patients with relapsed/refractory B cell acute lymphoblastic leukemia


Tisagenlecleucel is an autologous T cell genetically modified ex vivo using a lentiviral vector encoding an anti-CD19 chimeric antigen receptor. Here, we present the efficacy and safety of tisagenlecleucel in a subgroup of Japanese patients with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (ALL). ELIANA was a single-arm, open-label, multicenter, phase 2 study. Patients were aged ≥ 3 years at screening to ≤ 21 years at the time of diagnosis, and had ≥ 5% lymphoblasts in bone marrow at screening. Primary endpoint was overall remission rate [ORR; complete remission (CR) + CR with incomplete blood recovery (CRi)] within 3 months after infusion. As of April 13, 2018, eight patients were enrolled and six had been infused. ORR was 66.7% (95% confidence interval 22.3–95.7); three patients achieved CR and one patient had CRi. All patients with CR/CRi were negative for minimal residual disease. One patient had CR/CRi lasting 19.5 + months. Cytokine release syndrome (CRS) and neurological events occurred in 83% and 17% of patients, respectively. CRS resolved with anti-cytokine therapy and supportive care. Two deaths occurred due to disease progression. No cases of cerebral edema were observed. Tisagenlecleucel produced high remission rates and durable responses offering a new treatment option for Japanese pediatric and young adults with r/r B-ALL.

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We thank the patients and their families, the study investigators, and the study site personnel for their participations and contributions to this study. The current affiliation of Kazuto Natsume is AbbVie GK. We thank Rama Mylapuram (Novartis Healthcare Pvt. Ltd.) for medical editorial assistance with this paper.


This study was sponsored by Novartis Pharmaceuticals Corporation and Novartis Pharma K.K.

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Correspondence to Hidefumi Hiramatsu.

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Conflict of interest

IK reports research Grants from Novartis. SAG has received research and/or clinical trial support from Novartis, Servier and Kite. SAG reports consulting, member of study steering committees, or scientific/clinical advisory boards: Novartis, Cellectis, Adaptimmune, Eureka, TCR2, Juno, GlaxoSmithKline, Vertex, Cure Genetics, Humanigen, and Roche. LE, ACA, KN, KT, and YW are employees of Novartis. All other authors declare no competing interests.

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Hiramatsu, H., Adachi, S., Umeda, K. et al. Efficacy and safety of tisagenlecleucel in Japanese pediatric and young adult patients with relapsed/refractory B cell acute lymphoblastic leukemia. Int J Hematol 111, 303–310 (2020). https://doi.org/10.1007/s12185-019-02771-2

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  • Tisagenlecleucel
  • CAR T
  • Acute lymphoblastic leukemia
  • CTL019