NEJM:Asciminib二线治疗费城染色体阳性白血病

2019-12-12 MedSci MedSci原创

研究发现,Asciminib在先前靶向药物治疗失败的费城染色体阳性白血病患者中,表现出较高的活性。

Asciminib是BCR-ABL1蛋白变构抑制剂,其作用机制与其他ABL激酶抑制剂不同。近日研究人员评估了Asciminib在费城染色体阳性白血病患者中的安全性和抗白血病活性。

在剂量递增研究中,研究人员招募了141名处于慢性期和9名加速期慢性髓细胞白血病(CML)患者,患者先前接受了至少两种ATP竞争性酪氨酸激酶抑制剂(TKIs)治疗。研究的主要目的是确定Asciminib的最大耐受剂量或推荐剂量。患者每日一次或两次接受Aisminb治疗,剂量范围为10至200mg。中位随访时间为14个月。

150名患者参与研究,其中70%接受了至少3次TKI治疗。未达到Asciminib的最大耐受量。在慢性期CML患者中,34例(92%)的血液学复发患者有完全血液学反应;31例(54%)在基线时没有完全细胞遗传学反应的患者,在Asciminib治疗后获得完全的细胞遗传学反应。48%的患者在12个月内获得或维持了主要的分子学反应,其中包括8名(57%)对Ponatinib有抵抗或不可接受副作用的患者。5例(28%)T315I基因突变患者在12个月内获得或维持了主要的分子学反应。Asciminib治疗的临床反应持久,44例患者中有40例维持主要的分子学反应。剂量限制毒性作用包括脂肪酶水平无症状升高和临床胰腺炎。常见的不良反应包括疲劳、头痛、关节痛、高血压和血小板减少。

研究发现,Asciminib在先前靶向药物治疗失败的费城染色体阳性白血病患者中,表现出较高的活性。

原始出处:

Timothy P. Hughes et al. Asciminib in Chronic Myeloid Leukemia after ABL Kinase Inhibitor Failure. N Engl J Med, December 12, 2019.

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    2020-02-15 yanghan
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    2020-10-12 quxin068
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    2019-12-12 旺医

    顶刊就是顶刊,谢谢梅斯带来这么高水平的研究报道,我们科里同事经常看梅斯,分享梅斯上的信息

    0

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