文章：

慢性髓性白血病无治疗缓解策略与结局的真实世界证据

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

原文发布日期：26 June 2025

DOI: 10.3390/cancers17132148

类型: Article

开放获取: 是

英文摘要：

Background: 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.Methods: 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).Results: 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.Conclusions: Our study highlights the relative safety of pursuing TFR via different TKI treatment strategies in a real-world setting.

摘要翻译： 

背景：尽管酪氨酸激酶抑制剂（TKIs）疗效显著，但慢性髓系白血病（CML）患者常面临不良反应，这促使研究者探索对达到持续深度缓解的患者实施无治疗缓解（TFR）的可能性。方法：本研究旨在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的突然停药、停药前TKI剂量递减、或先进行TKI剂量降低随后在TFR前突然停药）。结果：我们检索了包含233名患者的CML数据库，确定了39名以TFR为目标的患者。中位TFR持续时间为14.6个月，其中63%的患者在21个月的中位随访期内仍处于TFR状态。在0-6个月、6-12个月、1-2年及>2年期间，分别有54%、16%、8%和21%的患者失去TFR。在三种TKI管理策略中，安全性结果相当，未出现疾病进展或CML相关死亡病例。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学反应（MMR）。在疗效方面，接受标准剂量TKI突然停药、标准剂量递减或预先剂量降低的患者中，分别有61%、59%和59%维持了TFR。结论：我们的研究强调了在真实世界环境中通过不同TKI治疗策略追求TFR的相对安全性。

原文链接：

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