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

被引:16
作者
Hiramatsu, Hidefumi [1 ]
Adachi, Souichi [2 ]
Umeda, Katsutsugu [1 ]
Kato, Itaru [1 ]
Eldjerou, Lamis [3 ]
Agostinho, Andrea Chassot [3 ]
Natsume, Kazuto [4 ]
Tokushige, Kota [4 ]
Watanabe, Yoko [4 ]
Grupp, Stephan A. [5 ,6 ]
机构
[1] Kyoto Univ, Grad Sch Med, Dept Pediat, Kyoto, Japan
[2] Kyoto Univ, Grad Sch Med, Human Hlth Sci, Kyoto, Japan
[3] Novartis Pharmaceut, E Hanover, NJ USA
[4] Novartis Pharma KK, Tokyo, Japan
[5] Childrens Hosp Philadelphia, Div Oncol, Philadelphia, PA 19104 USA
[6] Univ Penn, Dept Pediat, Perelman Sch Med, Philadelphia, PA 19104 USA
关键词
Tisagenlecleucel; CAR T; Acute lymphoblastic leukemia; ELIANA; CTL019; SURVIVAL; CHILDREN;
D O I
10.1007/s12185-019-02771-2
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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.
引用
收藏
页码:303 / 310
页数:8
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