This analysis from the multicenter, open-label, phase 3 BFORE trial reports efficacy and safety of bosutinib in patients with newly diagnosed chronic phase (CP) chronic myeloid leukemia (CML) after five years’ follow-up. Patients were randomized to 400-mg once-daily bosutinib (n = 268) or imatinib (n = 268; three untreated). At study completion, 59.7% of bosutinib- and 58.1% of imatinib-treated patients remained on study treatment. Median duration of treatment and time on study was 55 months in both groups. Cumulative major molecular response (MMR) rate by 5 years was higher with bosutinib versus imatinib (73.9% vs. 64.6%; odds ratio, 1.57 [95% CI, 1.08–2.28]), as were cumulative MR⁴ (58.2% vs. 48.1%; 1.50 [1.07–2.12]) and MR^4.5 (47.4% vs. 36.6%; 1.57 [1.11–2.22]) rates. Superior MR with bosutinib versus imatinib was consistent across Sokal risk groups, with greatest benefit seen in patients with high risk. Treatment-emergent adverse events (TEAEs) were consistent with 12-month data. After 5 years of follow-up there was an increase in the incidence of cardiac, effusion, renal, and vascular TEAEs in bosutinib- and imatinib-treated patients, but overall, no new safety signals were identified. These final results support 400-mg once-daily bosutinib as standard-of-care in patients with newly diagnosed CP CML.

This trial was registered at www.clinicaltrials.gov as #NCT02130557.

这项来自多中心、开放标签、3 期 BFORE 试验的分析报告了波舒替尼在五年随访后对新诊断的慢性期 (CP) 慢性粒细胞白血病 (CML) 患者的疗效和安全性。患者被随机分配至 400 毫克每日一次的波舒替尼（ n  = 268）或伊马替尼（n  = 268；三名未治疗）。研究完成时，59.7% 的波舒替尼治疗患者和 58.1% 的伊马替尼治疗患者仍在接受研究治疗。两组的中位治疗持续时间和研究时间均为 55 个月。博舒替尼与伊马替尼相比，5 年累积主要分子反应 (MMR) 率更高（73.9% 对 64.6%；优势比，1.57 [95% CI，1.08–2.28]），累积 MR ⁴也是如此（58.2% 对48.1%；1.50 [1.07–2.12]）和 MR ^4.5（47.4% 对 36.6%；1.57 [1.11–2.22]）率。在 Sokal 风险组中，波舒替尼与伊马替尼的优越 MR 一致，在高风险患者中获益最大。治疗中出现的不良事件 (TEAE) 与 12 个月的数据一致。经过 5 年的随访，波舒替尼和伊马替尼治疗的患者心脏、积液、肾脏和血管 TEAE 的发生率有所增加，但总体而言，没有发现新的安全信号。这些最终结果支持将 400 毫克每日一次的波舒替尼作为新诊断的 CP CML 患者的标准治疗。

该试验在 www.clinicaltrials.gov 注册为#NCT02130557。