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SOHO最新技术进展与后续问题 | 慢性髓性白血病(CML)无治疗缓解的最新情况

SOHO State of the Art Updates and Next Questions | Update on Treatment-Free Remission in Chronic Myeloid Leukemia (CML).

作者信息

Mikhaeel Sherrie, Atallah Ehab

机构信息

Medical College of Wisconsin, Milwaukee, WI.

Medical College of Wisconsin, Milwaukee, WI.

出版信息

Clin Lymphoma Myeloma Leuk. 2023 May;23(5):333-339. doi: 10.1016/j.clml.2023.02.008. Epub 2023 Feb 25.

Abstract

Tyrosine kinase inhibitor (TKI) discontinuation, also known as treatment-free remission (TFR) is currently one of the main goals of chronic myeloid leukemia (CML) therapy. TKI discontinuation should be considered in eligible patients for several reasons. Specifically, TKI therapy is associated with reduced quality of life, long-term side effects, and a heavy financial burden on both the patients and society. TKI discontinuation is a particularly important goal for younger patients diagnosed with CML because of the treatment's effects on their growth and development in addition to potential long-term side-effects. Numerous studies with thousands of patients have demonstrated the safety and feasibility of attempting TKI discontinuation in a select group of patients who have achieved a sustained deep molecular remission. With current TKIs, approximately 50% of patients will be eligible for attempting TFR of which only 50% will achieve a successful TFR. Therefore, in reality, only 20% of patients with newly diagnosed CML will achieve a successful TFR, and the majority of patients will need to continue TKI therapy indefinitely. However, several ongoing clinical trials are investigating treatment options for patients to achieve deeper remission with the ultimate goal of a cure, which is defined as being off drug with no evidence of disease.

摘要

酪氨酸激酶抑制剂(TKI)停药,也称为无治疗缓解(TFR),目前是慢性髓性白血病(CML)治疗的主要目标之一。由于多种原因,符合条件的患者应考虑停用TKI。具体而言,TKI治疗与生活质量下降、长期副作用以及患者和社会的沉重经济负担相关。对于诊断为CML的年轻患者,TKI停药是一个特别重要的目标,因为该治疗除了可能产生长期副作用外,还会影响他们的生长发育。针对数千名患者的大量研究表明,在一组实现持续深度分子缓解的特定患者中尝试停用TKI具有安全性和可行性。使用目前的TKI,约50%的患者将有资格尝试TFR,其中只有50%将实现成功的TFR。因此,实际上,新诊断的CML患者中只有20%将实现成功的TFR,大多数患者将需要无限期继续TKI治疗。然而,几项正在进行的临床试验正在研究患者实现更深缓解的治疗选择,最终目标是治愈,即停药且无疾病证据。

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