Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma
Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma
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DOI：
10.1182/bloodadvances.2020003092
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发表时间：
2020-11-10
期刊：
影响因子：
7.5
通讯作者：
Grupp, Stephan
中科院分区：
文献类型：
--
作者：
Pasquini, Marcelo C.;Hu, Zhen-Huan;Grupp, Stephan
Tisagenlecleucel是一种CD 19嵌合抗原受体（CAR）T细胞疗法，获批用于治疗儿童和年轻成人复发性/难治性急性淋巴细胞白血病（ALL）和成人非霍奇金淋巴瘤（NHL）。本文介绍了细胞治疗注册中心在真实环境中使用tisagenlecleucel的初步经验。截至2020年1月，73家中心共入组511例患者，报告了410例患者的随访数据（ALL，n = 255; NHL，n = 155），ALL和NHL的中位随访时间分别为13.4和11.9个月。在ALL患者中，初始完全缓解（CR）率为85.5%。10个月缓解持续时间（DOR）、无事件生存率和总生存率（OS）分别为60.9%、52.4%和77.2%。在NHL成人患者中，最佳总体缓解率为61.8%，包括39.5%的初始CR率。6个月DOR、无进展生存期和OS率分别为55.3%、38.7%和70.7%。在所有患者中，分别有11.6%和7.5%报告了≥ 3级细胞因子释放综合征和神经毒性。由于可行性，在使用符合质量标准和不符合质量标准产品的患者中观察到相似的结局
Tisagenlecleucel is a CD19 chimeric antigen receptor (CAR) T-cell therapy approved for treatment of pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia (ALL) and adults with non-Hodgkin lymphoma (NHL). The initial experience with tisagenlecleucel in a real-world setting from a cellular therapy registry is presented here. As of January 2020, 511 patients were enrolled from 73 centers, and 410 patients had follow-up data reported (ALL, n = 255; NHL, n = 155), with a median follow-up of 13.4 and 11.9 months for ALL and NHL, respectively. Among patients with ALL, the initial complete remission (CR) rate was 85.5%. Twelve-month duration of response (DOR), event-free survival, and overall survival (OS) rates were 60.9%, 52.4%, and 77.2%, respectively. Among adults with NHL, the best overall response rate was 61.8%, including an initial CR rate of 39.5%. Six-month DOR, progression-free survival, and OS rates were 55.3%, 38.7%, and 70.7%, respectively. Grade >= 3 cytokine release syndrome and neurotoxicity were reported in 11.6% and 7.5% of all patients, respectively. Similar outcomes were observed in patients with in-specification and out-of-specification products as a result of viability