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盐酸米托蒽醌脂质体联合阿糖胞苷治疗伴RUNX1∷MTG16融合基因的儿童急性髓系白血病:1例报告及文献复习

[Mitoxantrone hydrochloride liposome combined with cytarabine for treating pediatric acute myeloid leukemia with RUNX1∷MTG16 fusion gene: a case report and literature review].

作者信息

Lin S, Qi B Q, Liu L P, Xiao J G, Yang W Y, Zhu X F, Chen X J

机构信息

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin 300020, China Tianjin Institutes of Health Science, Tianjin 301600, China.

出版信息

Zhonghua Xue Ye Xue Za Zhi. 2024 Dec 14;45(12):1134-1137. doi: 10.3760/cma.j.cn121090-20240805-00289.

Abstract

This case report presents a patient with pediatric acute myeloid leukemia (AML) with RUNX1∷MTG16, admitted to the Blood Disease Hospital of the Chinese Academy of Medical Sciences in October 2023. He was 13 years old, with a chief complaint of fatigue for 20 days. Bone marrow smear revealed 17.0% blasts, the karyotype was 46,XY,t (16; 21) (q24; q22), molecular biology demonstrated RUNX1∷MTG16 fusion gene, combined with FLT3-ITD mutation. The child was diagnosed with AML (with RUNX1 ∷ MTG16). Complete remission was achieved after chemotherapy induction. The induction therapy regimen was mitoxantrone hydrochloride liposomes combined with cytarabine (MA). The RUNX1 ∷ MTG16 and FLT3-ITD were negative after another MA treatment course. However, the RUNX1 ∷ MTG16 and FLT3-ITD were turning positive during the following intensive treatment, and he then successfully underwent matched sibling donor umbilical cord blood transplantation.

摘要

本病例报告介绍了一名患有RUNX1∷MTG16的小儿急性髓系白血病(AML)患者,于2023年10月入住中国医学科学院血液病医院。患者为13岁男性,主要诉求为乏力20天。骨髓涂片显示原始细胞占17.0%,核型为46,XY,t(16;21)(q24;q22),分子生物学检测显示RUNX1∷MTG16融合基因,并伴有FLT3-ITD突变。该患儿被诊断为AML(伴RUNX1∷MTG16)。化疗诱导后达到完全缓解。诱导治疗方案为盐酸米托蒽醌脂质体联合阿糖胞苷(MA)。在另一个MA疗程后,RUNX1∷MTG16和FLT3-ITD均为阴性。然而,在随后的强化治疗期间,RUNX1∷MTG16和FLT3-ITD转为阳性,随后他成功接受了同胞全相合供者脐血移植。

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Design of liposomes as drug delivery system for therapeutic applications.脂质体作为治疗应用的药物传递系统的设计。
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