Wang Jing-Yu, Chen Jian-Fang, Wang Shi-Fang, Guo Qiao-Hua, Ma Yan-Ping
The Second Clinical Medical College of Shanxi Medical University; Taiyuan 030000, Shanxi Province, China.
Department of Hematology, The Second Hospital of Shanxi Medical University, Taiyuan 030000, Shanxi Province, China.
Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2023 Apr;31(2):476-482. doi: 10.19746/j.cnki.issn.1009-2137.2023.02.024.
To investigate the clinical characteristics, prognostic factors and efficacy of hypomethylating agent (HMA) in patients with chronic myelomonocytic leukemia (CMML).
The clinical data of 37 newly diagnosed patients with CMML was analyzed retrospectively, and their clinical characteristics and the efficacy of HMA were summarized. Kaplan-Meier and Log-rank test were used for univariate survival analysis, and Cox proportional hazards regression model was used for multivariate analysis.
The median age at diagnosis was 67 years old. Their common manifestations included fatigue, bleeding, abnormal blood routine and fever. Most patients had splenomegaly. According to FAB classification, there were 6 cases of myelodysplastic CMML and 31 cases of myeloproliferative CMML, while according to WHO classification, 8 patients belonged to CMML-0, 9 patients to CMML-1 and 20 patients to CMML-2. At the time of diagnosis, the median white blood cell count was 32.84×10/L, median hemoglobin (Hb) was 101 g/L, median platelet count was 65×10/L, median absolute monocyte count was 9.53×10//L, median absolute neutrophil count (ANC) was 11.29×10//L and median lactate dehydrogenase (LDH) was 374 U/L. Cytogenetic abnormalities were found in 4 cases among the 31 patients who underwent karyotype analysis or fluorescence in situ hybridization detection. There were 12 patients who had analyzable results and gene mutations were identified in 11 cases, including ASXL1, NRAS, TET2, SRSF2 and RUNX1. Among the 6 patients who were treated with HMA and could be evaluated for efficacy, 2 patients achieved complete remission, 1 patient achieved partial remission and 2 patients achieved clinical benefit. Compared with the non-HMA treatment group, overall survival (OS) time was not significantly prolonged in the HMA treatment group. Univariate analysis showed that Hb<100 g/L, ANC≥12×10/L, LDH≥250 U/L and peripheral blood (PB) blasts ≥5% were significantly associated with poor OS, while WHO classification CMML-2, Hb<100 g/L, ANC≥12×10/L, LDH≥250 U/L and PB blasts≥5% were significantly associated with poor leukemia-free survival (LFS) (P<0.05). Multivariate analysis showed that ANC≥12×10/L and PB blasts≥5% were significantly associated with poor OS and LFS (P<0.05).
CMML has high heterogeneity in clinical characteristics, genetic changes, prognosis and treatment response. HMA can not significantly improve the survival of CMML patients. ANC≥12×10/L and PB blasts≥5% are independent prognostic factors of OS and LFS in patients with CMML.
探讨慢性粒单核细胞白血病(CMML)患者的临床特征、预后因素及低甲基化药物(HMA)的疗效。
回顾性分析37例新诊断CMML患者的临床资料,总结其临床特征及HMA的疗效。采用Kaplan-Meier法和Log-rank检验进行单因素生存分析,采用Cox比例风险回归模型进行多因素分析。
诊断时的中位年龄为67岁。常见表现包括乏力、出血、血常规异常和发热。多数患者有脾肿大。根据FAB分类,骨髓增生异常型CMML 6例,骨髓增殖型CMML 31例;根据WHO分类,CMML-0 8例,CMML-1 9例,CMML-2 20例。诊断时,中位白细胞计数为32.84×10⁹/L,中位血红蛋白(Hb)为101 g/L,中位血小板计数为65×10⁹/L,中位绝对单核细胞计数为9.53×10⁹/L,中位绝对中性粒细胞计数(ANC)为11.29×10⁹/L,中位乳酸脱氢酶(LDH)为374 U/L。在31例行核型分析或荧光原位杂交检测的患者中,4例发现细胞遗传学异常。12例患者有可分析结果,11例检测到基因突变,包括ASXL1、NRAS、TET2、SRSF2和RUNX1。在6例接受HMA治疗且可评估疗效的患者中,2例达到完全缓解,1例达到部分缓解,2例获得临床获益。与非HMA治疗组相比,HMA治疗组的总生存(OS)时间未显著延长。单因素分析显示,Hb<100 g/L、ANC≥12×10⁹/L、LDH≥250 U/L和外周血(PB)原始细胞≥5%与OS不良显著相关,而WHO分类CMML-2、Hb<(此处原文有误,推测是100) g/L、ANC≥12×10⁹/L、LDH≥250 U/L和PB原始细胞≥5%与无白血病生存(LFS)不良显著相关(P<0.05)。多因素分析显示,ANC≥12×10⁹/L和PB原始细胞≥5%与OS和LFS不良显著相关(P<0.05)。
CMML在临床特征、基因改变、预后及治疗反应方面具有高度异质性。HMA不能显著提高CMML患者的生存率。ANC≥12×10⁹/L和PB原始细胞≥5%是CMML患者OS和LFS的独立预后因素。