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高危急性髓系白血病:当下及未来的治疗

High-risk acute myelogenous leukemia: treatment today ... and tomorrow.

作者信息

Schiller Gary J

机构信息

1Hematological Malignancy/Stem Cell Transplant Program, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA.

出版信息

Hematology Am Soc Hematol Educ Program. 2013;2013:201-8. doi: 10.1182/asheducation-2013.1.201.

Abstract

High-risk acute myelogenous leukemia (AML) constitutes a distinct subset of disease based on clinical and biological characteristics and comprises a significant percentage of all cases of adult AML. Biologic features such as distinct clonal cytogenetic and molecular abnormalities identify a subgroup of AML patients characterized by poor response to induction chemotherapy and poor long-term survival after treatment with consolidation chemotherapy. Clinical variables that predict for poor response include AML relapsed after less than 1 year of remission and AML characterized by resistance to conventional agents. We review here our understanding of the defining biologic subtypes of AML and discuss how adequate initial evaluation can be used to inform the choice of treatment. By defining high-risk biologic and clinical variables, a strong case can be made for treating patients with investigational agents, with treatment directed at distinct cytogenetic or molecular abnormalities. Allogeneic transplantation is the only form of therapy available outside of the setting of a clinical trial that may offer a chance for long-term survival for patients with high-risk AML.

摘要

高危急性髓系白血病(AML)基于临床和生物学特征构成了一个独特的疾病亚组,在所有成人AML病例中占相当大的比例。诸如独特的克隆细胞遗传学和分子异常等生物学特征确定了一组AML患者,其特点是对诱导化疗反应不佳,巩固化疗治疗后的长期生存率低。预测反应不佳的临床变量包括缓解期少于1年复发的AML以及对传统药物耐药的AML。我们在此回顾我们对AML定义性生物学亚型的理解,并讨论如何通过充分的初始评估来指导治疗选择。通过定义高危生物学和临床变量,可以有力地支持使用研究性药物治疗患者,针对不同的细胞遗传学或分子异常进行治疗。异基因移植是临床试验之外唯一可提供高危AML患者长期生存机会的治疗形式。

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