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慢性髓性白血病无治疗缓解策略及结果的真实世界证据

Real-World Evidence of Treatment-Free Remission Strategies and Outcomes in Chronic Myeloid Leukemia.

作者信息

Bourne Garrett, Diebold Kendall, Bascug Greg, Knapp Joshua, Espinoza-Gutarra Manuel, Vachhani Pankit, Bachiashvili Kimo, Rangaraju Sravanti, Mohty Razan, Bhatia Ravi, Jamy Omer

机构信息

Department of Medicine, University of Alabama at Birmingham, Birmingham, AL 35294, USA.

Division of Hematology and Oncology, Department of Medicine, University of Alabama at Birmingham, 1720 2nd Avenue S, NP2540W, Birmingham, AL 35294, USA.

出版信息

Cancers (Basel). 2025 Jun 26;17(13):2148. doi: 10.3390/cancers17132148.

Abstract

: Despite the remarkable efficacy of tyrosine kinase inhibitors (TKIs), patients with chronic myeloid leukemia (CML) often face adverse effects, prompting investigations into treatment-free remission (TFR) for patients with sustained deep responses. : Our objective was to assess real-world outcomes of TFR in a single-center cohort of patients in the southeastern U.S., as well as to compare different TKI management strategies (abrupt cessation of a TKI at a standard dose, TKI dose tapering prior to cessation, or upfront TKI dose reduction followed by abrupt cessation before TFR). : We queried our CML database of 233 patients and identified 39 patients that aimed for TFR. The median TFR duration was 14.6 months, with 63% actively remaining in TFR with a median follow-up of 21 m. TFR was lost by 54%, 16%, 8%, and 21% of patients in 0-6 m, 6-12 m, 1-2 y, and >2 y, respectively. Among the three TKI management strategies, the safety outcomes were comparable, with no instances of disease progression or CML-related mortality. All patients who lost TFR successfully regained a major molecular response (MMR) upon the resumption of TKIs. In terms of efficacy, 61%, 59%, and 59% of patients who underwent abrupt cessation of standard-dose TKIs, standard-dose tapering, or upfront dose reduction maintained TFR, respectively. : Our study highlights the relative safety of pursuing TFR via different TKI treatment strategies in a real-world setting.

摘要

尽管酪氨酸激酶抑制剂(TKIs)具有显著疗效,但慢性髓性白血病(CML)患者常面临不良反应,这促使人们对获得持续深度缓解的患者进行无治疗缓解(TFR)研究。我们的目的是评估美国东南部单中心队列中TFR的真实世界结果,并比较不同的TKI管理策略(标准剂量TKI突然停药、停药前逐渐减少TKI剂量,或在TFR前先减少TKI剂量然后突然停药)。我们查询了包含233例患者的CML数据库,确定了39例旨在实现TFR的患者。TFR的中位持续时间为14.6个月,63%的患者在中位随访21个月时仍处于TFR状态。分别有54%、16%、8%和21%的患者在0 - 6个月、6 - 12个月、1 - 2年和>2年时失去TFR。在三种TKI管理策略中,安全性结果相当,没有疾病进展或CML相关死亡的情况。所有失去TFR的患者在重新使用TKIs后均成功恢复了主要分子反应(MMR)。在疗效方面,标准剂量TKI突然停药、标准剂量逐渐减量或预先减量的患者中,分别有61%、59%和59%维持了TFR。我们的研究强调了在现实环境中通过不同TKI治疗策略追求TFR的相对安全性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/193e/12248582/16dbcdeb2f86/cancers-17-02148-g001.jpg

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