阿伐替尼治疗异基因造血干细胞移植后分子生物学阳性的伴KIT突变CBF-AML的疗效及安全性

The efficacy and safety of avapritinib in the treatment of molecular biologically positive core binding factor-acute myeloid leukemia with KIT mutation after allogeneic hematopoietic stem cell transplantation

目的探讨阿伐替尼治疗异基因造血干细胞移植(allo-HSCT)后分子生物学阳性的伴KIT突变核心结合因子相关性急性髓系白血病(CBF-AML)的疗效及安全性。方法纳入河南省肿瘤医院2021年12月至2023年3月收治的6例allo-HSCT后分子生物学阳性的伴KIT突变CBF-AML患者,均接受阿伐替尼治疗,回顾性分析阿伐替尼的疗效及安全性。结果阿伐替尼治疗1个月后,6例患者融合基因转录水平均较前下降,其中5例患者转录水平下降≥1 log。4例患者接受阿伐替尼治疗时间≥3个月,4例患者融合基因均转阴,转阴的中位时间为2.0(1.0~3.0)个月。至随访截止,4例患者无复发生存。最常见的不良反应是骨髓抑制,包括中性粒细胞减少2例、血小板减少2例、贫血1例。非血液学不良反应包括恶心2例、水肿1例、记忆力减退1例,均为1~2级。结论阿伐替尼可作为allo-HSCT后分子生物学阳性的伴KIT突变CBF-AML患者的有效治疗手段,主要的不良反应为骨髓抑制,患者耐受性良好。

Objective To investigate the efficacy and safety of avapritinib in the treatment of molecular biologically positive core binding factor-acute myeloid leukemia(CBF-AML)with KIT mutation after allogeneic hematopoietic stem cell transplantation(allo-HSCT).Methods We retrospectively analyzed the clinical data of six patients with molecular biologically positive CBF-AML with KIT mutation after allo-HSCT,who were treated with avapritinib at Henan Cancer Hospital from December 2021 to March 2023,and evaluated the efficacy and safety of avapritinib.Results After 1 month of treatment with avapritinib,the transcription level of the fusion gene decreased in six patients,and the transcription level decreased by≥1 log in five patients.In four patients who received avapritinib for≥3 months,the fusion gene turned negative,and the median time to turn negative was 2.0(range:1.0-3.0)months.Up to the end of follow-up,four patients had no recurrence.The most common adverse reaction of avapritinib was myelosuppression,including neutropenia in two cases,thrombocytopenia in two cases,and anemia in one case.The non-hematological adverse reactions were nausea in two cases,edema in one case,and memory loss in one case,all of which were grades 1-2.Conclusion Avapritinib was effective for molecular biologically positive CBF-AML patients with KIT mutation after allo-HSCT.The main adverse reaction was myelosuppression,which could generally be tolerated.