肿瘤细胞治疗研究
英文原题：Real-world Outcomes of Commercial Tisagenlecleucel for Children, Adolescents, and Young Adults With Acute Lymphoblastic Leukemia in Japan.
Real-world Outcomes of Commercial Tisagenlecleucel for Children, Adolescents, and Young Adults With Acute Lymphoblastic Leukemia in Japan.
tisagenlecleucel 在日本获批已有 5 年多。
嵌合抗原受体（CAR）T细胞疗法是复发/难治性（R/R）B细胞急性淋巴细胞白血病（B-ALL）儿童、青少年和年轻成人（CAYA）患者的重要新治疗选择。因此，积累世界各地区CAR-T真实世界疗效证据十分重要，尤其是比较医疗和族裔背景不同患者的结局。替沙格列赛在日本获批已超过5年。本文报告一项回顾性多机构研究，考察基线因素与临床结局之间的关联，旨在了解真实世界治疗经验并更充分评估商业化替沙格列赛的疗效。日本CAR-T联盟开展该项回顾性多中心研究，纳入接受商业化替沙格列赛CAR-T治疗的CAYA患者。分析包括42例R/R B-ALL患者，其白细胞单采样本已送至诺华进行商业化替沙格列赛制备。所有接受输注的患者均纳入应答、毒性和生存分析。最佳总体应答率为93%。输注后1年总生存率为82%，无事件生存率（EFS）为56%。输注替沙格列赛前，27例（64%）患者疾病负荷较低（LB，定义为骨髓[BM]淋巴母细胞<5%）。疾病负荷低与更佳结局相关：低负荷组1年EFS率为80%，而高负荷组（BM淋巴母细胞≥5%）为24%。多变量分析发现，既往接受造血干细胞移植（HSCT，n=23，55%）与较佳结局相关：既往HSCT患者1年EFS率为75%，未接受既往HSCT者为24%。这是日本首项分析接受商业化替沙格列赛治疗的R/R B-ALL CAYA患者研究，其疗效与临床试验及其他真实世界研究相近，并证实疾病负荷低及既往HSCT与更佳EFS相关。
Chimeric antigen receptor (CAR) T cells are a major new treatment option for children, adolescents, and young adults (CAYA) patients with relapsed and refractory (R/R) B cell acute lymphoblastic leukemia (B-ALL). Therefore, accumulating evidence from real-world experiences of CAR-T outcomes in various regions worldwide is important, particularly when comparing outcomes of patients with differing medical and ethnic backgrounds. More than 5 years have passed since tisagenlecleucel was approved in Japan. Here, we report a retrospective, multi-institutional investigation examining the association between baseline parameters and clinical outcomes. The aim was to investigate real-world experience and to better comprehend the efficacy of commercial tisagenlecleucel. A nationwide consortium called the Japan CAR-T Consortium conducted a retrospective, multicenter study of CAYA patients who received CAR-T cell treatment with commercial tisagenlecleucel. Forty-two patients with R/R B-ALL whose leukapheresis samples were shipped to Novartis for commercial tisagenlecleucel manufacture were included in the analysis. All infused patients were included in the response, toxicity, and survival analyses. The best overall response rate was 93%. The 1-year overall survival and event-free survival (EFS) rates after infusion were 82% and 56%, respectively. Twenty-seven (64%) had low disease burden (LB, defined as <5% bone marrow [BM] lymphoblasts) prior to tisagenlecleucel infusion. LB was associated with superior outcomes, with a 1-year EFS rate of 80% compared with 24% in high disease burden ( 5% BM lymphoblasts). Multivariate analysis identified an association between prior hematopoietic stem cell transplantation (HSCT) (n = 23, 55%) and superior outcomes, with a 1-year EFS rate of 75% compared with 24% for patients without prior HSCT. This first analysis of CAYA patients with R/R B-ALL undergoing treatment with commercial tisagenlecleucel in Japan reports an efficacy similar to that in clinical trials and other real-world studies and confirms that LB and prior HSCT are associated with superior EFS.
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