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米托蒽醌脂质体联合化疗方案治疗混合表型急性白血病的安全性及有效性研究

Safety and efficacy of mitoxantrone liposome combined chemotherapy in the treatment of mixed phenotype acute leukemia

摘要目的:分析米托蒽醌脂质体(MIT-LIP)联合化疗方案治疗混合表型急性白血病(MPAL)的安全性及有效性。方法:回顾性纳入2021年12月至2024年11月于华中科技大学同济医学院附属协和医院和黄冈市中心医院接受过MAED(MIT-LIP+阿糖胞苷+依托泊苷+地塞米松)方案的MPAL患者,收集患者的临床特点、不良反应、治疗效果、长期预后等数据。结果:共纳入7例接受过MAED方案化疗的MPAL患者,其中2例初始诊断分别为急性T淋巴细胞白血病和急性B淋巴细胞白血病,经过治疗后表型转换为急性髓系白血病,3例初始诊断为髓/B双克隆MPAL,1例初始诊断为髓/T双克隆MPAL,1例初始诊断为髓/浆细胞样树突状细胞双克隆MPAL。7例患者中,男3例,女4例;中位年龄38(16~58)岁;检测到染色体异常1例,基因异常6例,其中伴有BCR∷ABL融合基因1例。MAED方案化疗期间未发生明确相关的药物过敏及器官毒性,主要不良反应为血液系统毒性。经诱导缓解治疗后,所有患者均达到完全缓解(CR),2例患者仍处于巩固治疗中并维持微小残留病(MRD)阴性CR;1例患者维持MRD阳性CR;4例患者行异基因造血干细胞移植,2例维持MRD阴性CR,2例复发。目前中位随访时间为12个月,总生存(OS)率为100%,无复发生存(RFS)率为60%,中位OS时间和中位RFS时间未达到。结论:MAED方案治疗MPAL安全性良好,CR率高。

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abstractsObjective:To evaluate the safety and efficacy of mitoxantrone liposome (MIT-LIP) combined chemotherapy in treating mixed phenotype acute leukemia (MPAL) .Methods:December 2021 to November 2024, MPAL patients who underwent the MAED (MIT-LIP + cytarabine + etoposide + dexamethasone) regimen were retrospectively analyzed. Data on clinical characteristics, adverse reactions, therapeutic outcomes, and long-term prognoses were collected.Results:A total of 7 MPAL patients who received MAED regimen were admitted. Among them, two patients were initially diagnosed with T-ALL or B-ALL, respectively, and transformed into AML after treatment. Three patients were initially diagnosed as MPAL (B/myeloid), one as MPAL (T/myeloid), and one with MPAL (myeloid/plasmacytoid dendritic cell). Among the 7 patients, there were 3 males and 4 females, 1 chromosome abnormalities and 6 gene abnormalities, including 1 case with BCR∷ABL fusion gene. The median age was 38 years (range: 16–58 years). There was no clear related drug allergy and organ toxicity during MAED regimen, and the main adverse effect was hematological toxicity. After induced chemotherapy, all patients achieved complete remission (CR), 2 maintained MRD-negative CR and 1 maintained MRD-positive CR. The other 4 patients underwent allogeneic hematopoietic stem cell transplantation, 2 maintained MRD-negative CR, and 2 relapsed. The current median follow-up time was 12 months, the overall survival (OS) rate was 100%, the relapse-free survival (RFS) rate was 60%, and the median OS time and median RFS time were not reached.Conclusion:The MAED regimen demonstrates high safety and a favorable CR rate in MPAL treatment.

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