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CART 细胞治疗在儿童急性淋巴细胞白血病中的应用:面向普通儿科医师的综述

英文原题:CART-Cell Therapy in Pediatric Acute Lymphoblastic Leukemia: A Review for General Pediatricians.

PubMed 2026/09/10(内容时间) Pediatrics Q1 · IF 6.1(JCR 2025)

研究概要

嵌合抗原受体(CAR)T细胞疗法已经改变了复发/难治性CD19+ B细胞急性淋巴细胞白血病(B-ALL)儿童和年轻患者的结局,即使对化疗难治或造血干细胞移植(HSCT)后复发的患者也能实现深度缓解。

中文摘要

嵌合抗原受体(CAR)T细胞疗法已改变了复发/难治性CD19+ B细胞急性淋巴细胞白血病(B-ALL)儿童和年轻成人的结局,即使对化疗难治或造血干细胞移植(HSCT)后复发的患者,也能实现深度缓解。采用过继性细胞免疫治疗需要严格的临床路径,并在指定的CART细胞中心实施。初始完全缓解率高,且常为MRD(微小残留病)阴性,但复发仍较常见,原因包括持续性丧失或抗原逃逸。关键的急性毒性为细胞因子释放综合征(CRS)和免疫效应细胞相关神经毒性综合征(ICANS),而长期问题包括持续性血细胞减少、感染风险、B细胞再生障碍以及需要免疫球蛋白替代的低丙种球蛋白血症。本篇面向普通儿科医生的叙述性综述回顾了CART细胞治疗ALL的当前最新进展,并总结了关于CART细胞适应证、生产、结局、并发症以及与共享照护中心在短期和长期随访中相关问题的关键原则。

展开英文摘要原文

Chimeric antigen receptor (CAR) T cell therapy has transformed outcomes for children and young adults with relapsed/refractory CD19+ B-cell acute lymphoblastic leukemia (B-ALL), enabling deep remissions even in patients who are refractory to chemotherapy or have relapsed after hematopoietic stem cell transplantation (HSCT). Use of adoptive cellular immunotherapy requires a strict clinical pathway and is delivered at designated CART-cell centers. Initial complete remission rates are high and often MRD (minimal residual disease)-negative, but relapse remains frequent through loss of persistence or antigen escape. Key acute toxicities are cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), whereas longer-term issues include prolonged cytopenias, infection risk, B-cell aplasia, and hypogammaglobulinemia requiring immunoglobulin replacement. This narrative review for general pediatricians reviews the current state-of-the-art of CART-cell therapy for ALL and summarizes key principles about CART-cell indications, production, outcomes, complications, and relevant issues for shared-care centers on follow-up in the short and long term.

论文信息

作者
Grasso A、Mishra AK、Silva J、Amrolia PJ
单位
Department of Bone Marrow Transplantation, Great Ormond Street Children's Hospital, London, United Kingdom.United Kingdom
期刊
Pediatrics2026 Sep 10
原文标识
PubMed 42716532 · DOI 10.1542/peds.2026-076924