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儿童新发急性髓系白血病的临床挑战。

Clinical challenges in de novo pediatric acute myeloid leukemia.

机构信息

a Department of Pediatric Oncology/Hematology , VU University Medical Center , Amsterdam , The Netherlands.

b Dutch Childhood Oncology Group , The Hague , The Netherlands.

出版信息

Expert Rev Anticancer Ther. 2018 Mar;18(3):277-293. doi: 10.1080/14737140.2018.1428091. Epub 2018 Jan 20.

Abstract

Although the prognosis of pediatric acute myeloid leukemia (pAML) has improved, with current survival rates up to 75%, relapse rates remain high. Areas covered: The low number of patients, the heterogeneous genomic landscape of AML, novel diagnostic techniques, divergent available treatment protocols, and dose-limiting toxicity of conventional agents all contribute to the complexity of AML treatment. This review gives an overview of the current clinical challenges with respect to diagnostics, treatment, and supportive care in pAML. Expert commentary: Due to intensified treatment regimens and improved supportive care measures, the outcome for pAML patients has improved substantially over the past years. However, most treatment protocols still rely on conventional chemotherapeutic agents with significant toxicity. Although targeted therapies promise to reduce the need for high doses of conventional agents with a subsequent decrease in toxicity, the effectiveness of these strategies remains unsatisfactory today. International collaborations are needed in order to address the ongoing therapeutic challenges of reducing toxicity while increasing effectivity. Consensus on risk-group classification, a common chemotherapy backbone and evidence-based supportive care guidelines are necessary in this context, at the same time enabling intergroup studies on new agents in subgroups.

摘要

虽然儿科急性髓系白血病(pAML)的预后已经有所改善,目前的生存率高达 75%,但复发率仍然很高。

涵盖领域

患者数量少、AML 的基因组异质性景观、新的诊断技术、不同的可用治疗方案以及常规药物的剂量限制毒性,都增加了 AML 治疗的复杂性。这篇综述概述了 pAML 在诊断、治疗和支持性护理方面的当前临床挑战。

专家评论

由于强化治疗方案和改进的支持性护理措施,过去几年 pAML 患者的预后有了显著改善。然而,大多数治疗方案仍依赖于具有显著毒性的常规化疗药物。虽然靶向治疗有望减少对高剂量常规药物的需求,从而降低毒性,但这些策略的效果目前仍不尽人意。为了应对降低毒性同时提高疗效的持续治疗挑战,需要开展国际合作。在这种情况下,需要就风险组分类、共同的化疗基础和基于证据的支持性护理指南达成共识,同时能够在亚组中开展新药物的分组研究。

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