阿司米尼布是第一种特异性靶向ABL肌醇化袋（STAMP）的BCR-ABL1抑制剂，已获全球批准，用于治疗曾接受≥2种酪氨酸激酶抑制剂（TKIs）治疗的慢性期费城染色体阳性慢性髓细胞白血病成人患者（CML-CP）。在ASCEMBL试验中，接受≥2种先前TKIs治疗的CML-CP患者被随机分配（按基线主要细胞遗传学反应[MCyR]分层）为阿司米尼布40mg每日两次或博舒替尼500mg每日一次。与先前发表的主要分析结果一致，在2.3年的中位随访后，阿司米尼布继续展现出卓越的疗效和更好的安全性和耐受性，优于博舒替尼。在96周时的主要分子反应（MMR）率（关键次要终点）为阿司米尼布37.6％，而博舒替尼为15.8％;考虑到基线MCyR后，两组之间的MMR率差异为21.7％（95％ CI，10.53-32.95;双侧p = 0.001）。阿司米尼布出现的三级及以上不良事件（AEs）（56.4％与68.4％）和引起治疗中断的AEs（7.7％与26.3％）比博舒替尼少。相对于博舒替尼，阿司米尼布患者的治疗持续时间更长且继续获益，支持阿司米尼布作为曾接受≥2种TKIs治疗的CML-CP患者的标准治疗。©2023.作者。

Asciminib, the first BCR::ABL1 inhibitor that Specifically Targets the ABL Myristoyl Pocket (STAMP), is approved worldwide for the treatment of adults with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (CML-CP) treated with ≥2 prior tyrosine kinase inhibitors (TKIs). In ASCEMBL, patients with CML-CP treated with ≥2 prior TKIs were randomized (stratified by baseline major cytogenetic response [MCyR]) 2:1 to asciminib 40 mg twice daily or bosutinib 500 mg once daily. Consistent with previously published primary analysis results, after a median follow-up of 2.3 years, asciminib continued to demonstrate superior efficacy and better safety and tolerability than bosutinib. The major molecular response (MMR) rate at week 96 (key secondary endpoint) was 37.6% with asciminib vs 15.8% with bosutinib; the MMR rate difference between the arms, after adjusting for baseline MCyR, was 21.7% (95% CI, 10.53-32.95; two-sided p = 0.001). Fewer grade ≥3 adverse events (AEs) (56.4% vs 68.4%) and AEs leading to treatment discontinuation (7.7% vs 26.3%) occurred with asciminib than with bosutinib. A higher proportion of patients on asciminib than bosutinib remained on treatment and continued to derive benefit over time, supporting asciminib as a standard of care for patients with CML-CP previously treated with ≥2 TKIs.© 2023. The Author(s).