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首页> 外文期刊>Journal of Clinical Medicine >Pediatric AML: From Biology to Clinical Management
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Pediatric AML: From Biology to Clinical Management

机译：儿科AML：从生物学到临床管理

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Pediatric acute myeloid leukemia (AML) represents 15%–20% of all pediatric acute leukemias. Survival rates have increased over the past few decades to ~70%, due to improved supportive care, optimized risk stratification and intensified chemotherapy. In most children, AML presents as a de novo entity, but in a minority, it is a secondary malignancy. The diagnostic classification of pediatric AML includes a combination of morphology, cytochemistry, immunophenotyping and molecular genetics. Outcome is mainly dependent on the initial response to treatment and molecular and cytogenetic aberrations. Treatment consists of a combination of intensive anthracycline- and cytarabine-containing chemotherapy and stem cell transplantation in selected genetic high-risk cases or slow responders. In general, ~30% of all pediatric AML patients will suffer from relapse, whereas 5%–10% of the patients will die due to disease complications or the side-effects of the treatment. Targeted therapy may enhance anti-leukemic efficacy and minimize treatment-related morbidity and mortality, but requires detailed knowledge of the genetic abnormalities and aberrant pathways involved in leukemogenesis. These efforts towards future personalized therapy in a rare disease, such as pediatric AML, require intensive international collaboration in order to enhance the survival rates of pediatric AML, while aiming to reduce long-term toxicity.
机译：小儿急性髓细胞性白血病（AML）占所有小儿急性白血病的15％–20％。在过去的几十年中，由于改善了支持治疗，优化了风险分层和强化了化疗，使存活率提高到了约70％。在大多数儿童中，AML表现为新生，但在少数儿童中，AML是继发性恶性肿瘤。小儿AML的诊断分类包括形态学，细胞化学，免疫表型和分子遗传学的组合。结果主要取决于对治疗的初始反应以及分子和细胞遗传学畸变。在特定的遗传性高危病例或反应迟钝的患者中，治疗方法包括联合使用蒽环类和阿糖胞苷强化化疗和干细胞移植。通常，所有儿童AML患者中约30％会复发，而5％-10％的患者会由于疾病并发症或治疗的副作用而死亡。靶向治疗可增强抗白血病疗效，并最大程度降低与治疗相关的发病率和死亡率，但需要详细了解遗传异常和涉及白血病发生的异常途径。这些为将来在诸如AML等罕见疾病中进行个性化治疗的努力需要加强国际合作，以提高儿科AML的生存率，同时旨在降低长期毒性。

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