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儿童白血病不断演变的视野:新见解、挑战与未来之路

英文原题:Evolving Horizons in Pediatric Leukemia: Novel Insights, Challenges, and the Journey Ahead.

查看英文原题

Evolving Horizons in Pediatric Leukemia: Novel Insights, Challenges, and the Journey Ahead.

PubMed 2024/08/22(内容时间) Cureus

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中文摘要

儿童白血病,包括急性淋巴细胞白血病(ALL)和急性髓系白血病,尽管治疗取得了显著进展,仍然是一项艰巨的挑战。本综述通过全面分析近期文献,重点聚焦关键研究和临床试验,探讨了儿童白血病在免疫治疗、化疗和骨髓移植方面的最新进展。免疫治疗,包括单克隆抗体,如 blinatumomab 和 inotuzumab ozogamicin,以及CAR-T 细胞疗法,如 tisagenlecleucel 和 brexucabtagene autoleucel,已在复发/难治性 B 细胞 ALL(B-ALL)中展现出令人鼓舞的结果,达到了显著的缓解率且副作用可控。化疗仍然是主要治疗手段,采用根据个体风险特征量身定制的多阶段方案。骨髓移植,尤其是异基因干细胞移植,为高危或复发病例提供了潜在治愈可能,尽管其带来包括移植物抗宿主病和感染在内的风险。尽管取得了这些进展,治疗耐药、毒性和可及性问题依然存在。本综述还讨论了儿童白血病幸存者的长期结局,重点关注与化疗和骨髓移植等治疗相关的迟发性副作用,包括继发性恶性肿瘤、器官功能障碍和神经认知影响。持续的研究和临床试验对于完善这些疗法、提高其疗效并减少不良反应至关重要,最终改善年轻患者的生存率和生活质量。

展开英文摘要原文

Pediatric leukemia, encompassing acute lymphoblastic leukemia (ALL) and acute myeloid leukemia, remains a formidable challenge despite significant treatment advancements. This review examines recent developments in immunotherapy, chemotherapy, and bone marrow transplantation for pediatric leukemia through a comprehensive analysis of recent literature, focusing on critical studies and clinical trials. Immunotherapy, including monoclonal antibodies, such as blinatumomab and inotuzumab ozogamicin, and chimeric antigen receptor T-cell therapies, such as tisagenlecleucel and brexucabtagene autoleucel, have demonstrated promising results in relapsed or refractory B-cell ALL (B-ALL), achieving notable remission rates with manageable side effects. Chemotherapy continues to be the primary treatment, utilizing multiphase regimens tailored to individual risk profiles.

Bone marrow transplantation, especially allogeneic stem cell transplantation, offers potential cures for high-risk or relapsed cases, though it poses risks including graft-versus-host disease and infections. Despite these advancements, treatment resistance, toxicity, and accessibility persist.

This review also discusses the long-term outcomes among pediatric leukemia survivors, focusing on late-onset side effects associated with treatments such as chemotherapy and bone marrow transplantation, encompassing secondary malignancies, organ dysfunction, and neurocognitive impacts. Ongoing research and clinical trials are crucial to refine these therapies, enhance their efficacy, and reduce adverse effects, ultimately improving young patients' survival and quality of life.

论文信息

作者
Tito Rodriguez PR、Mehta D、Subhan M、Yadav RP、Yousofzai BS、Al-Najjar EH、Bibi R、Idries M
第一作者单位
Medicine, Universidad Privada del Valle, La Paz, BOL.United States
通讯作者单位
Pediatrics, Lady Reading Hospital, Peshawar, PAK.Pakistan
文献类型
综述
期刊
Cureus2024 Aug
原文标识
PubMed 39310608 · DOI 10.7759/cureus.67480