International Journal of Hematology

DOI: 10.1007/s12185-019-02771-2 Pages: 303-310

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

1. Kyoto University, Department of Pediatrics, Graduate School of Medicine

2. Kyoto University, Human Health Sciences, Graduate School of Medicine

3. Novartis Pharmaceuticals Co.

4. Novartis Pharma KK

5. Children’s Hospital of Philadelphia, Division of Oncology

6. University of Pennsylvania, Department of Pediatrics, Perelman School of Medicine

Correspondence to:
Hidefumi Hiramatsu



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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