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儿童急性淋巴细胞白血病治疗的转化进展:当前与新兴分子及免疫治疗的叙述性综述

英文原题:Translational advances in the treatment of childhood acute lymphoblastic leukemia: narrative review of current and emerging molecular and immunotherapies.

查看英文原题

Translational advances in the treatment of childhood acute lymphoblastic leukemia: narrative review of current and emerging molecular and immunotherapies.

PubMed 2023/02/24(内容时间) Transl Pediatr Q2 · IF 2(JCR 2025)

分数与星级只用于站内排序 —— 不代表疗效、安全性或个人适用性。

研究概要

尽管如此,仍需要更多的临床研究来进一步了解靶向治疗的潜在有害影响,并探究这些药物在治疗儿童 ALL 中替代或减少传统化疗使用的可能性。

中文摘要

急性淋巴细胞白血病(ALL)是儿童最常见的淋巴系血液恶性肿瘤。儿童新诊断ALL预后通常良好,5年OS超过90%;常规治疗已显著提高治愈率,但仍约三分之一患者复发或难治,构成儿童癌症相关死亡的重要原因。

作者广泛检索医学文献数据库,重点评估大型临床试验结果及重要血液学会议近期摘要。主要内容与发现:多数患者可通过现有治疗再诱导达到缓解,但长期总生存期仍不理想,构成治疗挑战。为持续改善儿童ALL结局,当前采用靶向分子治疗、免疫治疗和CAR-T 等新方法,以提高疗效并减少传统化疗副作用。这些方法利用早期基因组疾病特征分析及针对可操作分子靶点开发的药物。

仍需更多临床研究,以了解靶向治疗潜在危害,并评估其能否替代或减少儿童ALL常规化疗。

展开英文摘要原文

Acute lymphoblastic leukemia (ALL) is the most common hematologic malignancy of lymphoid origin in children. The prognosis for newly diagnosed ALL in the pediatric population is generally favorable, with a 5-year overall survival rate of more than 90%. Though conventional therapy has led to meaningful improvements in cure rates for new-onset pediatric ALL, one-third of patients still experience a relapse or refractory disease, contributing to a significant cause of pediatric cancer-related mortality.

An extensive literature review was undertaken via various databases of medical literature, focusing on both results of larger clinical trials, but also with evaluation of recent abstract publications at large hematologic conferences. KEY CONTENT AND FINDINGS: Remission is achievable in most of these patients by re-induction with currently available therapies, but the long-term overall survival rate is deemed suboptimal and remains a therapeutic challenge. As part of never-ceasing efforts to improve pediatric ALL outcomes, newer modalities, including targeted molecular therapies as well as immunotherapy, and chimeric antigen receptor (CAR) T-cell therapy, are currently being employed to increase treatment effectiveness as well as lessen the side effects from conventional chemotherapy. These approaches explore the use of early genome-based disease characterization and medications developed against actionable molecular targets.

Additional clinical research is nonetheless required to learn more about the potentially harmful effects of targeted therapies and investigate the possibility of these agents replacing or decreasing the use of conventional chemotherapy in treating pediatric ALL.

论文信息

作者
Munir F、He J、Connors J、Garcia M、Gibson A、McCall D、Nunez C、Dinh CN
单位
Department of Pediatrics, Pediatric Hematology Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.United States
文献类型
综述
期刊
Translational pediatrics2023 Mar 31
原文标识
PubMed 37035397 · DOI 10.21037/tp-22-656