维奈克拉联合阿扎胞苷序贯克拉屈滨治疗难治/复发儿童急性髓系白血病的近期疗效与安全性  

Safety and the Short-Term Efficacy of Venetoclax Combined with Azacitidine Followed by Cladribine in Children with Refractory/Relapsed Acute Myeloid Leukemia

作　　者：杜薇薇[1] 刘素香[1] 王易[1] 何海龙[1] 郭爱连 胡绍燕[1] 卢俊[1] DU Wei-Wei;LIU Su-Xiang;WANG Yi;HE Hai-Long;GUO Ai-Lian;HU Shao-Yan;LU Jun(Department of Hematology,Children's Hospital of Soochow University,Suzhou 215000,Jiangsu Province,China)

机构地区：[1]苏州大学附属儿童医院血液科,江苏苏州215000

出　　处：《中国实验血液学杂志》2023年第6期1635-1638,共4页Journal of Experimental Hematology

基　　金：国家自然科学基金项目(81970163、82170218);江苏省科技厅项目(BE2021654);江苏省卫健委项目(ZD2021006);苏州市科技计划项目(SS2019064);苏州市卫健委项目(GSWS2020039)。

摘　　要：目的:探讨维奈克拉联合阿扎胞苷序贯克拉屈滨(VAC方案)治疗难治/复发儿童急性髓系白血病的近期疗效与安全性。方法:回顾性分析2021年8月至2021年12月苏州大学附属儿童医院使用VAC方案治疗的6例难治/复发急性髓系白血病患儿的临床资料、治疗转归、并发症、血制品使用量。结果:6名患儿中,男1例,女5例;5例为难治AML,1例为复发的AML经异基因造血干细胞移植后16个月再次复发者;4例患儿伴有预后不良的染色体或基因,如RUNX1,FLT3-ITD,KMT2A exon 2-exon 8 dup,MLL-AF6,7q-,KMT2A exon 2-exon 10 dup等。6例患儿接受VAC方案治疗后,CR+CRi 4例、PR 1例(仅MRD未缓解,MRD为0.59%)、NR 1例(但骨髓原始细胞比例下降)。6例患儿均出现Ⅳ级中性粒细胞减少,4例出现Ⅳ级血小板减少。6例患儿在中性粒细胞缺乏期间均未出现发热、咳嗽、腹泻等感染症状。未发生治疗相关性死亡。结论:维奈克拉联合阿扎胞苷序贯克拉屈滨为早期诱导缓解效果不佳的复发/难治性AML患者提供了新的治疗选择,显示出较好的有效性和安全性。Objective:To investigate the safety and the short-term efficacy of venetoclax combined with azacitidine followed by cladribine(VAC regimen) in children with refractory/relapsed acute myeloid leukemia(AML).Methods:The clinical data,treatment outcomes,complications,and blood product consumption of 6 children with refractory/relapsed AML treated with VAC regimen in the Children's Hospital of Soochow University from August 2021 to December 2021 were retrospectively analyzed.Results:Among the 6 children,there were 1 male and 5 females.5 cases were refractory AML,and 1 case was relapsed AML,which recurred again 16 months after allogeneic hematopoietic stem cell transplantation.4 children were accompanied by chromosomes or genes that predicted poor prognosis,such as RUNX1,FLT3-ITD,KMT2A exon 2-exon 8 dup,MLL-AF6,7q-,KMT2A exon 2-exon 10 dup,etc.After received VAC regimen,4 cases achieved CR+CRi,1 case achieved PR(only MRD did not relieve,MRD was 0.59%),and 1 case was NR(but the proportion of bone marrow blasts decreased).All 6 patients had grade Ⅳ neutropenia,and 4 patients had grade Ⅳ thrombocytopenia.During the period of neutropenia,none of the 6 children developed symptoms of infection such as fever,cough,and diarrhea.No treatment-related death occurred.Conclusion:Venetoclax combined with azacitidine followed by cladribine provides a new treatment option for patients with relapsed/refractory AML who have poor efficacy in early induction remission theragy,showing good efficacy and safety.

关 键 词：维奈克拉 阿扎胞苷 克拉屈滨 儿童 急性髓系白血病 难治/复发 

分 类 号：R733.7[医药卫生—肿瘤]