摘要目的:分析慢性髓性白血病髓系急变期（CML-MBP）患者临床特征及总生存时间影响因素。方法:回顾性纳入2009年9月至2024年7月河南省肿瘤医院收治的CML-MBP患者，根据治疗方案的不同将患者分为联合组［确诊CML-MBP后使用酪氨酸激酶抑制剂（TKI）联合急性髓系白血病型方案治疗的患者］和单药组（使用单药TKI治疗的患者）。随访时间为自确诊CML-MBP起，至2024年10月16日，比较两组患者的临床特征，采用Cox回归模型分析患者生存时间的影响因素。结果:共纳入141例患者，联合组107例，男64例，女43例，年龄［ M（ Q1， Q3）］为43（32，52）岁；单药组34例，男19例，女15例，年龄51（45，61）岁。中位随访时间为6.5（2.5，15.1）个月。90.1%（127/141）的患者有慢性期/加速期病史，CML-MBP时，78.7%（111/141）的患者应用过TKI治疗。18.4%（26/141）的患者出现髓外急变，5.0%（7/141）为孤立性髓外急髓变。57.4%（81/141）的患者存在附加染色体异常，其中63.0%（51/81）为复杂染色体核型。58.0%（65/112）的患者艾贝尔森鼠白血病病毒癌基因同源物1（ABL1）激酶区突变阳性，T315I最常见。联合组年龄小于单药组，复杂染色体异常［63.6%（68/107）比38.2%（13/34）］、急变后应用三代TKI治疗［43.0%（46/107）比14.7%（5/34）］比例均高于单药组；白细胞计数［14.1（4.3，48.9）×10 9/L比50.1（11.5，149.0）×10 9/L］、血清乳酸脱氢酶水平［510（297，930）比900（535，1 898）U/L］、急变后应用伊马替尼治疗［8.4%（9/107）比20.5%（7/34）］的患者比例均低于单药组（均 P<0.05）。联合组和单药组分别有104和28例完成疗效评估，联合组主要分子学反应（MMR）率高于单药组［16.3%（17/104）比3.6%（1/28）， P=0.049］。联合组和单药组1年生存率（37.8%比32.4%， P=0.101）差异无统计学意义。多因素Cox回归模型分析显示：孤立性髓外急髓变（ HR=0.211，95% CI：0.064~0.704）、治疗获得主要血液学反应（MHR）（ HR=0.184，95% CI：0.102~0.330）和（或）深层分子学反应（DMR）（ HR=0.119，95% CI：0.028~0.504）是患者生存的保护因素。 结论:多数CML-MBP患者有慢性期/加速期病史和TKI治疗史，常伴复杂染色体核型和T315I突变，TKI联合治疗的患者MMR率较高，总体生存较差。孤立性髓外急髓变、治疗获得MHR和（或）DMR有利于CML-MBP患者的生存。

abstractsObjective:To analyze the clinical characteristics and factors influencing survival time in patients with chronic myeloid leukemia myeloid blast-phase (CML-MBP).Methods:The patients with CML-MBP treated at Henan Cancer Hospital from September 2009 to July 2024 were retrospectively enrolled. The patients were stratified into two groups based on different treatment regimens: the combination group [the patients receiving tyrosine kinase inhibitors (TKI) combined with an acute myeloid leukemia-type regimen following CML-MBP diagnosis] and the monotherapy group (the patients receiving TKI monotherapy). With follow-up extending from CML-MBP diagnosis until October 16, 2024, clinical characteristics were compared between groups. Potential factors influencing survival time were analyzed using Cox regression models.Results:A total of 141 patients were enrolled: 107 in the combination group [64 males and 43 females, aged M ( Q1, Q3), 43 (32, 52) years] and 34 in the monotherapy group [19 males and 15 females, aged 51 (45, 61) years]. The median follow-up duration was 6.5 (2.5, 15.1) months. About 90.1% (127/141) of the patients had a history of chronic/accelerated phase. During CML-MBP, 78.7% (111/141) of the patients had received TKI therapy. Extramedullary blast phase occurred in 18.4% (26/141) of the patients, with 5.0% (7/141) presenting as isolated extramedullary myeloid blast phase. Additional chromosomal abnormalities were detected in 57.4% (81/141) of the patients, of whom 63.0% (51/81) exhibited complex chromosomal karyotypes. Abelson murine leukemia viral oncogene homolog 1 (ABL1) kinase domain mutations were identified in 58.0% (65/112) of the patients, with the T315I mutation being the most common. The patients in the combination group were younger at diagnosis than those in the monotherapy group. The proportions of complex chromosomal abnormalities [63.6% (68/107) vs 38.2% (13/34)] and treatment with third-generation TKI following blast phase [43.0% (46/107) vs 14.7% (5/34)] were higher than those in the monotherapy group. The proportions of the patients with white blood cell count [14.1 (4.3, 48.9)×10?/L vs 50.1 (11.5, 149.0)×10?/L], serum lactate dehydrogenase level [510 (297, 930) vs 900 (535, 1 898) U/L], and treatment with imatinib following blast phase [8.4% (9/107) vs 20.5% (7/34)] were all lower than those in the monotherapy group (all P<0.05). Among 104 and 28 evaluable patients who completed efficacy evaluation in the combination group and monotherapy group, respectively, the major molecular response (MMR) rate was higher in the combination group [16.3% (17/104) vs 3.6% (1/28), P=0.049]. The 1-year survival rate did not differ significantly between the groups (37.8% vs 32.4%, P=0.101). Multivariate Cox regression analysis showed that isolated extramedullary myeloid blast phase ( HR=0.211, 95% CI: 0.064-0.704), achievement of major hematologic response (MHR) ( HR=0.184, 95% CI: 0.102-0.330), and/or deep molecular response (DMR) ( HR=0.119, 95% CI: 0.028-0.504) were identified as independent protective factors for survival. Conclusions:Most patients with CML-MBP have a history of chronic or accelerated phase disease and TKI therapy, frequently accompanied with complex chromosomal karyotypes and the T315I mutation. While patients receiving TKI combination therapy demonstrate higher rates of MMR, their overall survival remains poorer. Isolated extramedullary acute myeloid blast phase, and the achievement of MHR and/or DMR through treatment are beneficial for the survival of patients with CML-MBP.