无化疗方案治疗6例初治费城染色体阳性混合表型急性白血病的疗效分析  

Efficacy of chemo-free regimen in treatment of six patients with treatment-naive Philadelphia chromosome-positive mixed phenotype acute leukemia

作　　者：张莹[1] 范祎[1] 徐蜜蜜 屠雨青 相梦琪 仇惠英[1] 薛胜利[1] 唐晓文[1] 陈苏宁[1] 吴德沛[1] 陈佳[1] Zhang Ying;Fan Yi;Xu Mimi;Tu Yuqing;Xiang Mengqi;Qiu Huiying;Xue Shengli;Tang Xiaowen;Chen Suning;Wu Depei;Chen Jia(Department of Hematology,the First Affiliated Hospital of Soochow University,National Clinical Research Center for Hematologic Diseases,Jiangsu Institute of Hematology,Suzhou 215000,China)

机构地区：[1]苏州大学附属第一医院血液科,国家血液系统疾病临床医学研究中心,江苏省血液研究所,苏州215000

出　　处：《中华医学杂志》2025年第9期708-712,共5页National Medical Journal of China

基　　金：国家重点研发计划(2022YFC2502700);国家自然科学基金(82370215);江苏省血液病医学创新中心(CXZX202201);苏州市医学应用基础研究(SKY2023047)。

摘　　要：分析无化疗方案治疗初治费城染色体(Ph)阳性混合表型急性白血病(Ph+MPAL)的疗效。回顾性纳入2021年7月1日至2023年10月31日在苏州大学附属第一医院接受维奈克拉(VEN)、阿扎胞苷(AZA)联合酪氨酸激酶抑制剂(TKIs)组成的无化疗方案治疗的初治Ph+MPAL患者的临床资料。末次随访时间为2024年12月1日。分析患者的完全缓解/血细胞计数未完全恢复的完全缓解(CR/CRi)、微小残留病(MRD)、生存及安全性情况。共纳入6例患者,男1例,女5例,年龄40(25~52)岁,初诊时外周血白细胞为191.4(13.6~344.0)×10^(9)/L。6例患者均为B系和髓系混合表达。5例患者检测到Ph染色体,其中3例可见附加染色体异常。6例患者在1疗程的诱导治疗后均获得治疗反应,其中3例达CR,3例达CRi,且6例患者均达到骨髓MRD阴性。治疗1.5(0.4~5.9)个月后,6例患者均达到分子学缓解(MMR)。2例患者出现复发。5例患者接受异基因造血干细胞移植治疗。随访时间31.4(13.1~40.6)个月,6例患者均处于无病生存状态。严重不良反应主要为血液学毒性和败血症,无治疗相关死亡事件的发生。VEN+AZA+TKIs组成的无化疗方案治疗初治Ph+MPAL的疗效较好。To investigate the efficacy of chemo-free regimen in treatment of patients with treatment-naive Philadelphia chromosome positive mixed phenotype acute leukemia(Ph+MPAL).The clinical data of patients with newly treated Ph+MPAL who received venetoclax(VEN),azacytidine(AZA)and tyrosine kinase inhibitors(TKIs)in the First Affiliated Hospital of Soochow University from July 1,2021 to October 31,2023 were retrospectively included.The last follow-up date was December 1,2024.The complete remission/complete remission with incomplete blood count recovery(CR/CRi),measurable residual disease(MRD),survival and safety were analyzed.A total of 6 patients were included,with 1 male and 5 females,aged 40(25-52)years.The peripheral white blood count at diagnosis was 191.4(13.6-344.0)×10^(9)/L.All 6 patients had mixed expression of B lineage and myeloid lineage.Philadelphia chromosome was detected in 5 patients,and 3 of them had additional chromosomal abnormalities.All 6 patients achieved response after 1 cycle induction therapy,including 3 patients who achieved CR and 3 patients who achieved CRi.And all 6 patients attained bone marrow MRD negativity.After treatment of 1.5(0.4-5.9)months,6 patients achieved molecular remission(MMR).Tow patients experienced recurrence.Five patients received allogeneic hematopoietic stem cell transplantation.The follow-up time was 31.4(13.1-40.6)months,and all 6 patients were in disease-free survival.Severe adverse events mainly included hematological toxicity and sepsis,and no treatment-related deaths occurred.The VEN+AZA+TKIs chemo-free regimen has good efficacy in treating newly diagnosed Ph+MPAL patients.

关 键 词：白血病 双表型 急性 费城染色体 无化疗 

分 类 号：R73[医药卫生—肿瘤]