分子监测作为治疗急性早幼粒细胞白血病的途径。

Rare cancers and therapy Pub Date : 2015-01-01 Epub Date: 2015-10-21 DOI:10.1007/s40487-015-0013-8
Federico De Angelis, Massimo Breccia
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引用次数: 7

摘要

急性早幼粒细胞白血病(APL)是一种分子明确的疾病,以特异性染色体易位为特征;生物学和临床知识的提高以及随后引入的分子靶向治疗已经改变了APL的管理,目前存活率超过80%。APL最小残留病(MRD)评估是其治疗的最重要工具;早幼粒细胞白血病视黄酸受体α (PML-RARα)的分子检测在巩固治疗后早期识别血液学复发中的预后作用现已得到证实,并指导先发制人的治疗。最初使用定性聚合酶链反应(PCR)方法进行的经验被更准确的实时定量PCR (RQ-PCR)所取代,这保证了MRD的数字定量。三氧化二砷(ATO)不仅在复发患者中是有效的治疗方法,而且可以作为单独或与全反式维甲酸联合标准治疗的替代疗法,考虑到创新疗法与标准疗法可能对PML-RARα的清除率不同,这扩大了APL患者MRD评估的验证范围。MRD监测在自体和异体干细胞移植或使用其他生物制剂的复发患者中也显示了其有效性。这篇综述的目的是报告和讨论APL中MRD的实际现状。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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Molecular Monitoring as a Path to Cure Acute Promyelocytic Leukemia.

Acute promyelocytic leukemia (APL) is a molecularly well-defined disease, characterized by a specific chromosomal translocation; the improvement in biologic and clinical knowledge and subsequent introduction of molecularly targeted therapies have transformed the management of APL, with survival rates now exceeding 80%. Minimal residual disease (MRD) assessment in APL is the most important tool for its treatment; the prognostic role of the molecular detection of promyelocytic leukemia retinoic acid receptor α (PML-RARα) transcript after consolidation therapy in the early identification of the following hematologic relapse is now well established and guides preemptive therapy. First experiences performed with a qualitative polymerase chain reaction (PCR) approach were replaced with more accurate real-time quantitative PCR (RQ-PCR), which guarantees a numeric quantification of MRD. The identification of arsenic trioxide (ATO) as a valid therapy not only in relapsed patients but also as an alternative to standard therapy alone or in association with all-trans-retinoic acid enlarges the setting of validation of MRD evaluation in APL patients, considering a possible different clearance of PML-RARα with innovative therapy different from the standard ones. MRD monitoring demonstrated its validity also in the setting of relapsed patients with interesting results in the autologous and allogeneic stem cell transplantation setting or with the use of other biological agents. The aim of this review is to report and discuss the actual state of the art of MRD in APL.

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