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单中心66例慢性粒单核细胞白血病(CMML)患者的治疗结果及预后因素

Treatment Outcomes and Prognostic Factors in 66 Patients with Chronic Myelomonocytic Leukemia (CMML) in a Single Center.

作者信息

Wang Chao, Wang Zhiqiong, Meng Fankai, Luo Li, Liu Xian, Shi Jiayu, Huang Lifang

机构信息

Department of Hepatic Surgery, Institute of Hepato-Pancreato-Biliary Surgery, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030, People's Republic of China.

Department of Hematology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030, People's Republic of China.

出版信息

Int J Gen Med. 2022 Oct 18;15:7843-7854. doi: 10.2147/IJGM.S371766. eCollection 2022.

Abstract

INTRODUCTION

Chronic myelomonocytic leukemia (CMML) is a rare hematological malignancy bearing of both myelodysplastic syndrome and myeloproliferative neoplasm characteristics. Despite the low incidence, the clinical diagnosis of CMML was difficult and the survival was poor. The optimal first-line therapy for CMML still remains a matter of debate.

METHODS

We retrospectively analyzed the clinical characteristics of 66 CMML patients in a single center during the past 10 years and studied the survival status of CMML patients in the real world and the influence of treatment methods on the prognosis of patients.

RESULTS

For the 66 CMML patients, the median age was 60 years old (IQR 47.0-67.0), and an approximately 1.6:1.0 male-to-female ratio was found. CMML-0, CMML-1 and CMML-2 accounted for 13.7% (9/66), 43.9% (29/66) and 42.4% (28/66), respectively. The chromosome abnormality rate was 27.2% (18/66). Gene mutation was detected in 60 patients by sequenced in first-generation with 51.1% (22/43) gene mutations and in NGS with 82.3% (14/17) gene mutations. The top three mutation genes were (11/60, 18.3%), (10/60, 16.7%), and (9/60, 15.0%). There were 27 patients in supportive therapy group, and 39 patients in chemotherapy group including patients undergoing HSCT. Patients in chemotherapy group showed better OS than those in the supportive group before and after PSM analysis with p < 0.05. Patients with blast cell in bone marrow ≥10% or WHO CMML-2 benefited more from chemotherapy treatment achieving better OS. Multivariate analysis showed that supportive therapy and intermediate-2/high in CPSS were independent risk factors for OS after PSM.

DISCUSSION

Chemotherapy including hypomethylating agents prolonged overall survival of CMML patients, especially in patients with blast cell ≥10% in bone marrow or WHO CMML-2 comparing with supportive therapy. Sequencing may provide direct insight into the molecular mechanism by detection of gene mutation, enabling personalized treatment in the future.

摘要

引言

慢性粒单核细胞白血病(CMML)是一种罕见的血液系统恶性肿瘤,兼具骨髓增生异常综合征和骨髓增殖性肿瘤的特征。尽管发病率较低,但CMML的临床诊断困难,生存率较差。CMML的最佳一线治疗方案仍存在争议。

方法

我们回顾性分析了单中心过去10年中66例CMML患者的临床特征,研究了CMML患者在现实世界中的生存状况以及治疗方法对患者预后的影响。

结果

66例CMML患者的中位年龄为60岁(四分位间距47.0 - 67.0),男女比例约为1.6:1.0。CMML-0、CMML-1和CMML-2分别占13.7%(9/66)、43.9%(29/66)和42.4%(28/66)。染色体异常率为27.2%(18/66)。通过一代测序在60例患者中检测到基因突变,基因突变率为51.1%(22/43),通过二代测序检测到基因突变率为82.3%(14/17)。前三位突变基因分别为(11/60,18.3%)、(10/60,16.7%)和(9/60,15.0%)。支持治疗组有27例患者,化疗组有39例患者,包括接受造血干细胞移植的患者。倾向评分匹配(PSM)分析前后,化疗组患者的总生存期(OS)均优于支持治疗组,p < 0.05。骨髓原始细胞≥10%或世界卫生组织(WHO)CMML-2的患者从化疗中获益更多,OS更好。多因素分析显示,PSM后支持治疗和CPSS中2/高危是OS的独立危险因素。

讨论

包括去甲基化药物在内的化疗延长了CMML患者的总生存期,尤其是骨髓原始细胞≥10%或WHO CMML-2的患者,与支持治疗相比。测序可通过检测基因突变直接洞察分子机制,有望在未来实现个性化治疗。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c686/9835103/ffdf0094a97e/IJGM-15-7843-g0001.jpg

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