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启发的研讨会第4A部分:在B细胞急性淋巴细胞白血病的独特人群中获得CAR T细胞治疗

Lena E Winestone¹, Deepa Bhojwani², Sara Ghorashian³

¹Division of Allergy, Immunology, and BMT, Department of Pediatrics, UCSF Benioff Children's Hospitals, UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, California.

Transplantation and cellular therapy

|October 11, 2023

概括

在初步试验之外,tisagenlecleucel (CAR-T) 治疗对B-ALL患者有希望,包括中枢神经系统复发患者. 现实数据现在支持在罕见和高风险的儿科B-ALL群体中扩大使用.

科学领域:

在瘤学瘤学.
免疫治疗是一种免疫疗法.
儿科血液学儿科血液学

背景情况:

基于ELIANA试验,Tisagenlecleucel (tisa-cel) 已被批准用于儿科B细胞急性淋巴细胞白血病 (B-ALL).
ELIANA试验排除了特定的患者亚组,并且缺乏足够的数量用于罕见的亚群.
提萨细胞的商业化为更广泛的应用产生了现实世界数据 (RWD) 和临床试验数据.

研究的目的:

评估CD19 CAR-T治疗在特殊人群和儿科B-ALL的罕见临床情景中的疗效和安全性.
解决Tisa-cel标签未涵盖的适用于疾病的数据缺口,包括中枢神经系统复发和极高风险患者.
探索早期CD19CAR-T在高风险B-ALL群体中使用的潜在好处.

主要方法:

对现实世界的数据和临床试验数据的综合分析.
对中枢神经系统 (CNS) 复发性疾病患者的结果的综述.
评估CD19 CAR-T在患有持续性最小残留疾病 (MRD) 和第一次复发的患者中的使用.

主要成果:

现实数据支持CD19 CAR-T在中枢神经系统复发性疾病中的疗效,这与之前有关神经毒性的担忧相反.
正在出现的数据是早期使用在非常高风险的患者,包括那些持久的MRD和第一次复发.
高风险人群,如婴儿,21型综合症患者和年轻人,可能会从早期的CD19 CAR-T治疗中受益.

结论:

现实世界和临床数据扩大了对特殊人群中儿科B-ALLCD19CAR-T治疗的理解.
需要进一步的前性研究来评估患者报告的结果和这种新型治疗方法的获取差异.

关键词:

获得护理服务的机会.细胞疗法是一种细胞疗法.细胞遗传学细胞遗传学淋巴细胞白血病 (Lymphoblastic Leukemia) 是一种淋巴细胞白血病.患者报告的结果.

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