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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

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。

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abstractsTo 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.

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中华医学杂志

中华医学杂志

2025年105卷9期

708-712页

MEDLINEISTICPKUCSCDCA

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