北京中医药大学学报2025，Vol.48Issue(9)：1211-1221,11.DOI:10.3969/j.issn.1006-2157.2025.09.005
U2AF1突变骨髓增生异常综合征患者的生存特征及含砷中药复方的临床疗效研究
Survival characteristics of myelodysplastic syndrome with U2AF1-mutation and the clinical efficacy of arsenic-containing traditional Chinese medicine compound prescription
摘要
Abstract
Objective To explore the clinical characteristics of myelodysplastic syndrome(MDS)with U2 small nuclear RNA cofactor 1(U2AF1)mutation in different age groups,as well as the efficacy and prognosis of an arsenic-containing traditional Chinese medicine(TCM)compound prescription(Qinghuang Capsules combined with Bushen Yijing Formula).Methods A retrospective analysis was conducted on the clinical data of patients with MDS who were hospitalized in the Hematology Department Ward of Xiyuan Hospital,China Academy of Chinese Medical Sciences,and received arsenic-containing TCM compound treatment from November 30,2020,to September 30,2023.Stratified by age,the U2AF1 mutation and wild-type groups aged＜65 years and≥65 years were compared in terms of sex,TCM syndrome,World Health Organization classification,MDS Revised International Prognostic Score System(IPSS-R)score,blood routine indicators,serum lactate dehydrogenase content,nephroblastoma 1(WT1)expression level,bone marrow puncture and biopsy indicators,and chromosomal prognostic grades,et al.Furthermore,the efficacy of arsenic-containing TCM compound were compared in the U2AF1 mutation and wild-type groups among different age groups,as well as the influence of age on the survival prognosis of MDS patients with U2AF1 mutation.Results A total of 201 patients with MDS were included.104 patients were under 65 years old,among whom 20 had U2AF1 mutation,and 84 had wild-type.Ninety-seven patients were aged 65 years or older,among whom 19 patients had the U2AF1 mutation and 78 had the wild-type.Among patients aged＜65 years,the U2AF1 mutation group had a higher proportion of male patients and very low-risk/low-risk patients with an IPSS-R score≤3(P＜0.05),a lower mean corpuscular volume(MCV)(P＜0.05),and a relatively higher proportion of peripheral blood cell line 1 reduction than the wild-type group(P＜0.05).Among patients aged≥65 years,the MCV in the U2AF1 mutation group was lower(P＜0.05),and the expression level of the bone marrow WT1 gene and the proportion of patients with reticular fiber grade 4 were relatively higher than in the wild-type group(P＜0.05).The total effective rate of the arsenic-containing TCM compound for patients with U2AF1 mutation was 61.5％(24/39),and the total response rate was 30.8％(12/39).The total effective rate for the wild-type patients was 67.9％(110/162),and the total response rate was 29.6％(48/162).No significant difference was observed in the total effective and response rates.Kaplan-Meier survival analysis of 39 patients with U2AF1 mutation revealed that the median overall survival(mOS)of patients older than 65 years had not been reached.The 1-,2-,and 3-year survival rates were 93.8％,84.4％,and 84.4％,respectively.The mOS of the patients aged≥65 years was 35 months(95％confidence interval[CI]:7.559-62.441),and the 1-,2-,and 3-year survival rates were 66.2％,58.9％,and 29.4％,respectively.The mOS of patients in the aged≥65 years group was significantly lower than that in the aged＜65 years group(P＜0.05),and no significant difference was observed in median progression-free survival between the two groups.Conclusion The U2AF1 mutation is closely associated with the clinical characteristics of MDS.However,age and the presence of U2AF1 mutation have no significant effect on the total effective and response rates of arsenic-containing TCM compound.Age is a significant factor influencing the prognosis of patients with MDS with U2AF1 mutation.Patients aged 65 years or older have a shorter survival time than those younger than 65 years.关键词
U2小核RNA辅助因子1突变/骨髓增生异常综合征/髓毒劳/青黄散/补肾益精方/生存分析/回顾性研究Key words
U2 small nuclear RNA cofactor 1 mutation/myelodysplastic syndrome/myelotoxic fatigue/Qinghuang Powder/Bushen Yijing Formula/survival analysis/retrospective study分类
医药卫生引用本文复制引用
张朝畅,麻柔,唐旭东,刘驰,王德秀,陈卓,吕妍,肖海燕,刘为易,许勇钢,杨秀鹏..U2AF1突变骨髓增生异常综合征患者的生存特征及含砷中药复方的临床疗效研究[J].北京中医药大学学报,2025,48(9):1211-1221,11.基金项目
国家自然科学基金面上项目(No.82074258,No.82274502) （No.82074258,No.82274502）
第二批北京市研究型病房示范建设项目(No.BCRW202108) （No.BCRW202108）
中国中医科学院科技创新工程重大攻关项目(No.CI2021A01701) National Natural Science Foundation of China(Nos.82074258 and 82274502) （No.CI2021A01701）
