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实现慢性髓性白血病深度分子反应和无治疗缓解的当前策略及未来方向

Current Strategies and Future Directions to Achieve Deep Molecular Response and Treatment-Free Remission in Chronic Myeloid Leukemia.

作者信息

Annunziata Mario, Bonifacio Massimiliano, Breccia Massimo, Castagnetti Fausto, Gozzini Antonella, Iurlo Alessandra, Pregno Patrizia, Stagno Fabio, Specchia Giorgina

机构信息

Hematology Division, AORN Cardarelli-Napoli, Naples, Italy.

Section of Hematology, Department of Medicine, University of Verona, Verona, Italy.

出版信息

Front Oncol. 2020 Jun 2;10:883. doi: 10.3389/fonc.2020.00883. eCollection 2020.

Abstract

The treatment of chronic myeloid leukemia (CML) has been radically changed by the approval of tyrosine kinase inhibitors (TKIs), which target BCR-ABL1 kinase activity. CML is now managed as a chronic disease requiring long-term treatment and close molecular monitoring. It has been shown that in a substantial number of patients who have achieved a stable deep molecular response (DMR), TKI treatment can be safely discontinued without loss of response. Therefore, treatment-free remission (TFR), through the achievement of a DMR, is increasingly regarded as a feasible treatment goal in many CML patients. However, only nilotinib has approval in this setting and a number of controversial aspects remain regarding treatment choices and timings, predictive factors, patient communication, and optimal strategies to achieve successful TFR. This narrative review aims to provide a comprehensive overview on how to optimize the path to DMR and TFR in patients with CML, and discusses recent data and future directions.

摘要

酪氨酸激酶抑制剂(TKIs)的获批从根本上改变了慢性髓性白血病(CML)的治疗方式,这类抑制剂可靶向作用于BCR-ABL1激酶活性。如今,CML被当作一种需要长期治疗和密切分子监测的慢性病来管理。研究表明,在大量已实现稳定深度分子反应(DMR)的患者中,可以安全地停用TKI治疗且不会丧失疗效。因此,通过实现DMR来达到无治疗缓解(TFR),在许多CML患者中越来越被视为一个可行的治疗目标。然而,在此情况下只有尼罗替尼获得了批准,在治疗选择和时机、预测因素、患者沟通以及实现成功TFR的最佳策略等方面仍存在一些争议。本叙述性综述旨在全面概述如何优化CML患者实现DMR和TFR的路径,并讨论最新数据和未来方向。

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