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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患者的疗效

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

作者信息

Zhang Y, Fan Y, Xu M M, Tu Y Q, Xiang M Q, Qiu H Y, Xue S L, Tang X W, Chen S N, Wu D P, Chen J

机构信息

Department of Hematology, the First Affiliated Hospital of Soochow University, National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, Suzhou 215000, China.

出版信息

Zhonghua Yi Xue Za Zhi. 2025 Mar 4;105(9):708-712. doi: 10.3760/cma.j.cn112137-20241029-02423.

Abstract

To investigate the efficacy of chemo-free regimen in treatment of patients with treatment-naive Philadelphia chromosome positive mixed phenotype acute leukemia(PhMPAL). The clinical data of patients with newly treated PhMPAL 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/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 PhMPAL patients.

摘要

探讨无化疗方案治疗初治费城染色体阳性混合表型急性白血病(PhMPAL)患者的疗效。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)和酪氨酸激酶抑制剂(TKIs)治疗的初治PhMPAL患者的临床资料。末次随访日期为2024年12月1日。分析完全缓解/血细胞计数未完全恢复的完全缓解(CR/CRi)、微小残留病(MRD)、生存率和安全性。共纳入6例患者,其中男性1例,女性5例,年龄40(25 - 52)岁。诊断时外周血白细胞计数为191.4(13.6 - 344.0)×10⁹/L。6例患者均有B系和髓系混合表达。5例患者检测到费城染色体,其中3例有额外的染色体异常。6例患者在1周期诱导治疗后均获得缓解,其中3例达到CR,3例达到CRi。6例患者均实现骨髓MRD阴性。经过1.5(0.4 - 5.9)个月的治疗,6例患者实现分子缓解(MMR)。2例患者复发。5例患者接受了异基因造血干细胞移植。随访时间为31.4(13.1 - 40.6)个月,6例患者均无病生存。严重不良事件主要包括血液学毒性和败血症,未发生与治疗相关的死亡。VEN + AZA + TKIs无化疗方案治疗新诊断的PhMPAL患者疗效良好。

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