治疗急性髓系白血病新药：Hedgehog信号通路抑制剂glasdegib

A new drug in treating acute myeloid leukemia: Hedgehog signaling pathway inhibitor glasdegib

急性髓系白血病(AML)是成人常见的急性白血病。Hedgehog信号通路的异常与AML的发病密切相关。glasdegib(Gla)为一种Hedgehog信号通路抑制剂,由辉瑞制药公司生产,于2018年11月由美国食品和药物管理局批准上市,与低剂量阿糖胞苷联合用于治疗新确诊的75岁及以上或因其他合并症而无法接受高强度化疗的AML患者。临床试验表明,Gla联合低剂量阿糖胞苷可明显改善患者的生存期。最常见的不良反应包括贫血、疲劳、出血、发热、中性粒细胞下降、肌肉疼痛、血小板减少和呼吸困难等。

Acute myeloid leukemia(AML)is common acute leukemia in adults.Abnormalities in the Hedgehog signaling pathway are closely related to the pathogenesis of AML.Glasdegib(Gla)is a Hedgehog pathway inhibitor developed by Pfizer Inc.and approved by the U.S.Food and Drug Administration in November 2018.It is indicated,in combination with low-dose cytarabine,for the treatment of newly-diagnosed AML in adult patients who are≥75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.Clinical trials have showed that Gla combined with low-dose cytarabine can significantly improve the survival of patients.The most common adverse reactions are anemia,fatigue,bleeding,fever,neutropenia,muscle pain,thrombocytopenia,dyspnea,and so on.