文章：

慢性髓性白血病临床治疗中断的可行性：来自全国236例患者系列研究的结果

Feasibility of treatment discontinuation in chronic myeloid leukemia in clinical practice: results from a nationwide series of 236 patients

原文发布日期：2018-12-02

DOI: 10.1038/s41408-018-0125-0

类型: Article

开放获取: 是

英文摘要：

Over half of chronic myeloid leukemia (CML) patients in deep molecular response do not lose the major molecular response (MMR) after stopping treatment with tyrosine kinase inhibitors (TKI). This strategy is safe in clinical trials, but its applicability in the real-life setting remains unsettled. We describe the outcomes after TKI discontinuation in a nationwide series of 236 CML patients. Median follow-up from treatment discontinuation was 21.5 months and 5 patients died from CML-unrelated causes. TKI therapy was reinitiated due to MMR loss (n = 52), increase ≥ 1 log in BCR-ABL transcript level without losing MMR (n = 12), patient preference (n = 2), and withdrawal syndrome (n = 1). Treatment-free remission rate at 4 years was 64% (95% confidence interval, CI: 55%–72%). Cumulative incidence of molecular recurrence at 3 years was 33% (95% CI: 26%–38%). TKI treatment for < 5 years and MR4.5 duration shorter than 4 years were both associated with higher incidence of molecular recurrence. No patient had disease progression. Response status at last control was: MR4.5 (n = 196), MR4 (n = 15), MMR (n = 14), complete cytogenetic response (n = 10), and other (n = 1). A significant increase in Hb and cholesterol levels was observed after imatinib withdrawal. Our results demonstrate that TKI treatment discontinuation is feasible in real-life clinical practice.

摘要翻译： 

超过半数的达到深度分子学反应的慢性髓系白血病（CML）患者在停止酪氨酸激酶抑制剂（TKI）治疗后并未丧失主要分子学反应（MMR）。该策略在临床试验中被证实是安全的，但在实际临床环境中的适用性仍待明确。我们通过全国性队列研究分析了236例CML患者中止TKI治疗后的结局。治疗中止后的中位随访时间为21.5个月，其中5例患者因非CML原因死亡。重启TKI治疗的原因包括：丧失MMR（52例）、BCR-ABL转录水平升高≥1对数级但未丧失MMR（12例）、患者意愿（2例）及戒断综合征（1例）。4年无治疗缓解率为64%（95%置信区间CI：55%-72%），3年分子学复发累积发生率为33%（95%CI：26%-38%）。TKI治疗时长<5年及MR4.5持续时间短于4年均与较高的分子学复发率相关。所有患者均无疾病进展。末次随访时的反应状态为：MR4.5（196例）、MR4（15例）、MMR（14例）、完全细胞遗传学反应（10例）及其他（1例）。伊马替尼停药后观察到血红蛋白和胆固醇水平显著升高。本研究结果证实TKI治疗中止在实际临床实践中具有可行性。

原文链接：

Feasibility of treatment discontinuation in chronic myeloid leukemia in clinical practice: results from a nationwide series of 236 patients