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酪氨酸激酶抑制剂在慢性髓性白血病治疗中的停药:对当前实践的批判性评价。

Tyrosine kinase inhibitor discontinuation in the management of chronic myeloid leukemia: a critical review of the current practice.

机构信息

Hematology, Department of Translational and Precision Medicine, Azienda Policlinico Umberto I, Sapienza University , Rome, Italy.

Italian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit , Rome, Italy.

出版信息

Expert Rev Hematol. 2020 Dec;13(12):1311-1318. doi: 10.1080/17474086.2021.1852924. Epub 2020 Dec 1.

DOI:10.1080/17474086.2021.1852924
PMID:33205694
Abstract

: Tyrosine kinase inhibitors (TKIs), which target BCR-ABL1 kinase activity, have significantly prolonged the overall survival of patients affected by chronic myeloid leukemia (CML) and changed drastically the outcome. Evidences from several studies suggest that in patients who have achieved a sustained, stable and deep molecular response, TKI treatment can be safely discontinued with a close subsequent monitoring. Thus, a stable deep molecular response (DMR) has become a feasible treatment goal in CML. : In this review, the main findings extrapolated from sponsored and real-life evidences regarding TKI discontinuation were discussed, through a broad research on Medline, Embase and archives from EHA and ASH congresses (including words such as discontinuation, treatment-free remission, TFR, etc). Moreover, suggestions emerged from international guidelines about treatment-free remission (TFR) are presented. : With the growing availability of clinical trials and real-life data on TFR, in recent years the possibility of offering to CML patients a safe, informed and shorter path to TFR, through the achievement of a stable deep molecular response (DMR), has become an increasing option. However, many controversial aspects remain regarding treatment choices and timings, predictive factors, patient communication and optimal strategies aimed at achieving a successful TFR.

摘要

酪氨酸激酶抑制剂(TKIs),靶向 BCR-ABL1 激酶活性,显著延长了慢性髓性白血病(CML)患者的总生存期,并彻底改变了其预后。多项研究的证据表明,在达到持续、稳定和深度分子缓解的患者中,TKI 治疗可以在密切监测后续情况的情况下安全停药。因此,稳定的深度分子缓解(DMR)已成为 CML 的一种可行治疗目标。

在这篇综述中,通过对 Medline、Embase 和 EHA 及 ASH 大会档案的广泛研究(包括停药、无治疗缓解、TFR 等词),讨论了从赞助和真实世界证据中得出的关于 TKI 停药的主要发现。此外,还介绍了关于无治疗缓解(TFR)的国际指南中的建议。

随着 TFR 的临床试验和真实数据的不断增加,近年来,通过实现稳定的深度分子缓解(DMR),为 CML 患者提供安全、知情和更短的 TFR 途径已成为越来越多的选择。然而,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,仍存在许多争议。

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