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临床试验之外的慢性髓性白血病患者的酪氨酸激酶抑制剂能够停药吗?

Can any patients with chronic myeloid leukemia outside of a clinical trial have their tyrosine kinase inhibitor discontinued?

作者信息

Mauro Michael J

机构信息

Memorial Sloan Kettering Cancer Center, New York, New York, USA.

出版信息

Curr Opin Hematol. 2017 Mar;24(2):125-131. doi: 10.1097/MOH.0000000000000321.

Abstract

PURPOSE OF REVIEW

This article critically appraises the state of treatment-free remission as a strategy for patients with chronic myeloid leukemia (CML) in deep remission after therapy with tyrosine kinase inhibitors (TKIs).

RECENT FINDINGS

Approximately half of patients with CML defined fairly narrowly by trial criteria - TKI sensitive, in deep molecular remission for a defined period - can successfully maintain protective levels of response after TKI cessation. Those who cannot appear at very low risk of disease control loss and can promptly regain remission with TKI resumption. Increasing numbers of patients followed longer term in trials have proven as well as a lack of additional late relapse in either group and that 'functional cure' of CML is feasible. Both the definition of remission sufficient to attempt treatment-free remission and the trigger to resume treatment have been relaxed somewhat while outcomes have remained the same. Based on repeated confirmatory data, economic pressures, and pragmatism, the question of feasibility and safety of TKI cessation outside of clinical trials is at hand.

SUMMARY

TKI cessation outside of clinical trials, if performed under strict guidelines, utilizing optimal monitoring techniques, with counsel available from experts in the field, and after full disclosure of the risks and benefits with the patient, may be safe (see video, supplemental digital content 1, which summarizes the abstract and offers the author's perspective,http://links.lww.com/COH/A15).

摘要

综述目的

本文批判性地评估了对于接受酪氨酸激酶抑制剂(TKI)治疗后处于深度缓解期的慢性髓性白血病(CML)患者,停止治疗实现缓解作为一种治疗策略的现状。

最新发现

按照试验标准较为严格定义的CML患者中,约一半——对TKI敏感、在特定时期处于深度分子缓解状态——在停止TKI治疗后能够成功维持具有保护作用的缓解水平。那些无法维持缓解的患者疾病控制丧失风险极低,重新使用TKI后可迅速恢复缓解。越来越多在试验中接受更长时间随访的患者已得到证实,而且两组均未出现额外的晚期复发情况,CML的“功能性治愈”是可行的。在结果保持不变的情况下,足以尝试停止治疗实现缓解的缓解定义以及重新开始治疗的触发因素都有所放宽。基于反复的验证数据、经济压力和务实态度,临床试验之外停止使用TKI的可行性和安全性问题已摆在面前。

总结

在临床试验之外停止使用TKI,如果在严格的指导原则下进行,采用最佳监测技术,有该领域专家提供咨询,并在向患者充分披露风险和益处之后,可能是安全的(见视频,补充数字内容1,总结了摘要并提供了作者的观点,http://links.lww.com/COH/A15)。

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