This final analysis from the phase 4 BYOND trial reports outcomes with bosutinib in patients with previously treated chronic myeloid leukemia (CML); 163 patients with CML resistant/intolerant to previous tyrosine kinase inhibitors received bosutinib (starting dose: 500 mg QD). At study completion (median follow-up, 47.8 months), 48.1% (n = 75/156) of patients with Philadelphia chromosome–positive chronic phase CML were still receiving treatment. Among evaluable patients, 71.8% (95% CI, 63.9–78.9) and 59.7% (95% CI, 51.4–67.7) attained or maintained major molecular response (MMR) and molecular response (MR)⁴, respectively, at any time on treatment. The majority of patients achieved a deeper molecular response relative to baseline while on bosutinib. Kaplan-Meier probabilities (95% CI) of maintaining MMR and MR⁴ at 36 months were 87.2% (78.0–92.7) and 80.7% (69.4–88.1), respectively. At 48 months, the Kaplan-Meier overall survival rate was 88.3% (95% CI, 81.8–92.6); there were 17 deaths, including 2 that were considered CML related. Long-term adverse events (AEs) were consistent with the known safety profile of bosutinib, and no new safety issues were identified. The management of AEs through dose reduction maintained efficacy while improving tolerability. These results support the use of bosutinib in patients with previously treated CML.

ClinicalTrials.gov, NCT02228382

来自 4 期 BYOND 试验的最终分析报告了 bosutinib 在既往接受过治疗的慢性粒细胞白血病 （CML） 患者中的结局;163 例对既往酪氨酸激酶抑制剂耐药/不耐受的 CML 患者接受了博舒替尼治疗（起始剂量：500 mg QD）。在研究完成时 （中位随访，47.8 个月），48.1% （n = 75/156） 的费城染色体阳性慢性期 CML 患者仍在接受治疗。在可评估的患者中，71.8% （95% CI， 63.9-78.9） 和 59.7% （95% CI， 51.4-67.7） 在治疗的任何时间分别达到或维持主要分子反应 （MMR） 和分子反应 （MR）⁴。大多数患者在服用 bosutinib 期间实现了相对于基线更深的分子反应。在 36 个月时维持 MMR 和 MR⁴ 的 Kaplan-Meier 概率 （95% CI） 分别为 87.2% （78.0-92.7） 和 80.7% （69.4-88.1）。48 个月时，Kaplan-Meier 总生存率为 88.3% （95% CI，81.8-92.6）;有 17 例死亡，包括 2 例被认为与 CML 相关。长期不良事件 （AEs） 与 bosutinib 的已知安全性一致，未发现新的安全性问题。通过减少剂量来管理 AE 在提高耐受性的同时保持了疗效。这些结果支持在既往接受过治疗的 CML 患者中使用 bosutinib。

ClinicalTrials.gov、NCT02228382