PDF(Pubmed)
Abstract：
Asciminib is a first-in-class BCR::ABL1 inhibitor that Specifically Targets the ABL1 Myristoyl Pocket (STAMP). It is approved worldwide and in Japan for chronic myeloid leukemia in chronic phase (CML-CP) with resistance or intolerance to previous tyrosine kinase inhibitor (TKI) therapy. In the Phase 3 ASCEMBL study, patients with CML-CP who received ≥ 2 prior ATP-competitive TKIs were randomized (2:1) to asciminib 40 mg twice-daily or bosutinib 500 mg once-daily. Here, we report the 96-week results of the subgroup analysis of Japanese patients (asciminib, n = 13; bosutinib, n = 3) in the ASCEMBL study. The MMR rate at Week 96 was 46.2% in asciminib-treated patients, increasing from Weeks 24 and 48. Patients who achieved MMR at Week 24 remained in MMR up to the Week 96 cutoff. While a high proportion of patients treated with asciminib remained on treatment at cutoff, none randomized to bosutinib were on treatment at Week 96. Despite the longer duration of exposure to asciminib, its safety and tolerability continued to be favorable with no new or worsening safety findings. Overall, the efficacy and safety outcomes in the Japanese subgroup were comparable with the ASCEMBL global study population, which supports the use of asciminib in Japanese patients with previously treated CML-CP.
摘要：
Asciminib是一类BCR::ABL1抑制剂，其特异性靶向ABL1肉豆蔻酰口袋（STAMP）。它在全球和日本被批准用于慢性期慢性髓性白血病（CML-CP），对以前的酪氨酸激酶抑制剂（TKI）治疗具有抗性或不耐受。在第三阶段ASCEMBL研究中，接受过2次以上ATP竞争性TKIs治疗的CML-CP患者被随机分组(2:1),分别接受阿司替尼40mg,每日2次或博舒替尼500mg,每日1次.这里,我们报告了日本患者亚组分析的96周结果(阿西替尼，n=13；博舒替尼，n=3）在ASCEMBL研究中。在接受阿西替尼治疗的患者中，第96周的MMR率为46.2％，从第24周和第48周开始增加。在第24周达到MMR的患者保持MMR直至第96周截止值。虽然接受阿西替尼治疗的患者比例很高，但仍在接受治疗。在第96周,没有随机接受博舒替尼治疗.尽管阿西替尼暴露时间较长，其安全性和耐受性继续良好,没有新的或恶化的安全性发现.总的来说,日本亚组的疗效和安全性结果与ASCEMBL全球研究人群相当，这支持在先前治疗过的CML-CP的日本患者中使用阿西替尼。
