The aim of this multicenter phase 2 trial, Stop Nilotinib (NILSt), was to examine the safety and efficacy of discontinuation of nilotinib in patients with chronic phase (CP)-chronic myelogenous leukemia (CML). Patients with CP-CML who had achieved molecular response (MR4.5) after initiation of imatinib or nilotinib therapy received consolidation therapy with nilotinib 300-400 mg twice daily for up to 24 months. Patients who maintained MR4.5 at 24 months of consolidation therapy proceeded to discontinuation of nilotinib. The study enrolled 149 patients; 112 patients proceeded to consolidation therapy with nilotinib; 90 patients maintained MR4.5 with consolidation therapy, and 87 proceeded to discontinuation of nilotinib. The treatment-free remission (TFR) (MR4.5) rate at both 1 and 3 years after discontinuation of nilotinib was the same, at 60.9% (90% CI 51.6-69.7). Among 34 patients with molecular relapse, nilotinib was resumed in 33 patients; all of them attained MR4.5. There was no significant association between molecular relapse and age, sex, Sokal score, previous interferon-α exposure, duration of tyrosine kinase inhibitors treatment, or trough concentration of nilotinib. With nilotinib, it might be possible to avoid prognostic factors for TFR that exist with imatinib discontinuation. Cessation of nilotinib after two years of consolidation was safe and feasible.Trial registration UMIN000007141.

中文翻译：

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慢性粒细胞性白血病患者停用尼罗替尼并保持深度分子反应 2 年：一项多中心 2 期试验，停用尼罗替尼 (NILSt)。

这项多中心 2 期试验 Stop Nilotinib (NILSt) 的目的是检查在慢性期 (CP)-慢性粒细胞白血病 (CML) 患者中停用尼罗替尼的安全性和有效性。在伊马替尼或尼罗替尼治疗开始后达到分子反应 (MR4.5) 的 CP-CML 患者接受尼罗替尼 300-400 mg 每天两次的巩固治疗长达 24 个月。在巩固治疗 24 个月时维持 MR4.5 的患者继续停用尼罗替尼。该研究招募了 149 名患者；112 名患者使用尼罗替尼进行巩固治疗；90 名患者通过巩固治疗维持 MR4.5，87 名患者继续停用尼罗替尼。尼罗替尼停药后 1 年和 3 年的无治疗缓解率 (TFR) (MR4.5) 相同，均为 60。9%（90% CI 51.6-69.7）。34例分子复发患者中，33例恢复尼罗替尼；他们都达到了MR4.5。分子复发与年龄、性别、Sokal 评分、先前的干扰素-α 暴露、酪氨酸激酶抑制剂治疗的持续时间或尼罗替尼的谷浓度之间没有显着关联。使用尼罗替尼，有可能避免伊马替尼停药时存在的 TFR 预后因素。巩固两年后停用尼罗替尼安全可行。试验注册UMIN000007141。酪氨酸激酶抑制剂治疗的持续时间，或尼罗替尼的谷浓度。使用尼罗替尼，有可能避免伊马替尼停药时存在的 TFR 预后因素。巩固两年后停用尼罗替尼安全可行。试验注册UMIN000007141。酪氨酸激酶抑制剂治疗的持续时间，或尼罗替尼的谷浓度。使用尼罗替尼，有可能避免伊马替尼停药时存在的 TFR 预后因素。巩固两年后停用尼罗替尼安全可行。试验注册UMIN000007141。