在阶段 II 的开放标签 DIALOG 研究中，验证了尼洛替尼在免疫耐受（R/I）或新诊断（ND）的幼儿期 Philadelphia 染色体阳性慢性髓系白血病慢性期（CML-CP）患者中的疗效和安全性。在这份最终分析中，呈现了治疗完成 66 个周期（每周期 28 天）的尼洛替尼（剂量为每天 230 毫克/平方米，分两次服用）或提前停药患者的长期疗效和安全性。共招募了 59 名患者，其中 58 名接受了治疗（R/I 群体为 33 名，ND 群体为 25 名；两个群体的中位治疗时间分别为 60.5 个月和 51.9 个月）。在 R/I 群体中，累计发生主要分子应答（MMR）的比率为 60.6%，没有患者出现确认的 MMR 丧失。在 ND 患者中，最佳整体 MMR 比率为 76.0%，有三名患者有确认的 MMR 丧失。通过 66 个周期，R/I 群体的累计分子应答 MR4 和 MR4.5 比率（BCR::ABL1IS≤0.01% 和≤0.0032%，分别）分别为 27.3% 和 12.1%，而 ND 群体则分别为 56.0% 和 44.0%。尼洛替尼的安全性与先前报告一致，没有治疗期间的死亡事件发生。这些长期（约 5 年）的数据支持尼洛替尼在幼儿期 Ph+ CML-CP 患者中的疗效和安全性。www.clinicaltrials.gov.uk #NCT01844765. 版权所有 © 2023 年美国血液学会。

The efficacy and safety of nilotinib in pediatric patients with imatinib/dasatinib resistant/intolerant (R/I) or newly diagnosed (ND) Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia in chronic phase (CML-CP) was demonstrated in the phase II, open-label DIALOG study. In this final analysis, long-term efficacy and safety are presented for patients who completed 66 cycles (of 28 days) of treatment with nilotinib (230 mg/m2 twice daily) or discontinued early. Fifty-nine patients were enrolled and 58 were treated (R/I, n=33; ND n=25; median time on treatment: 60.5 and 51.9 months, respectively). In the R/I cohort, the cumulative major molecular response (MMR) rate was 60.6% and no patients had a confirmed loss of MMR. Among ND patients, the best overall MMR rate was 76.0%; three patients had a confirmed loss of MMR. The cumulative molecular response MR4 and MR4.5 (BCR::ABL1IS ≤0.01% and ≤0.0032%, respectively) rates by 66 cycles were 27.3% and 12.1% in the R/I cohort, and 56.0% and 44.0% in the ND cohort, respectively. The safety profile of nilotinib was consistent with earlier reports. No on-treatment deaths occurred. These long-term (up to approximately 5 years) data support the efficacy and safety of nilotinib in pediatric patients with Ph+ CML-CP. www.clinicaltrials.gov.uk #NCT01844765.Copyright © 2023 American Society of Hematology.