Citation: | YAN Zonghai, WU Yanming, DING Gang, LI Chengjun, SUN Guangyu. Effect of hypomethylating agents in the treatment of intermediate-and high-risk myelodysplasia syndrome[J]. Journal of Clinical Medicine in Practice, 2024, 28(5): 75-78, 84. DOI: 10.7619/jcmp.20233639 |
To observe the clinical effect of hypomethylating agents (HMAs) in the treatment of patients with intermediate- and high-risk myelodysplastic syndrome (MDS).
A retrospective study was conducted in 58 patients with intermediate-and high-risk MDS. The study group(25 patients) received azacitidine or decitabine for hypomethylating treatment, while the control group(33 patients) received routine symptomatic supportive treatment. The clinical efficacy, hematologic parameters, quality of life, and adverse events were observed and compared between the two groups.
After treatment, the complete remission rate, objective response rate, and disease control rate were higher in the study group than in the control group, while the disease progression rate was lower (P < 0.05). The 1-year survival rate was 92.00% in the study group, which was higher than 75.76% in the control group, but the difference showed no statistically significant (P>0.05). After treatment, hemoglobin, white blood cell, and platelet levels were higher in both groups than before treatment, and were higher in the study group than in the control group (P < 0.05). The total incidence of adverse events was lower in the study group than that in the control group (P < 0.05). After treatment, the quality of life scores (physical function, cognitive function, emotional function, role function, and social function) were higher in the study group than those in the control group (P < 0.05).
HMAs treatment is superior to routine symptomatic supportive treatment in patients with intermediate-and high-risk MDS, and not only improves the quality of life but also has good safety.
[1] |
PATNAIK M M, TEFFERI A. Myelodysplastic syndromes with ring sideroblasts (MDS-RS) and MDS/myeloproliferative neoplasm with RS and thrombocytosis (MDS/MPN-RS-T) -"2021 update on diagnosis, risk-stratification, and management"[J]. Am J Hematol, 2021, 96(3): 379-394. doi: 10.1002/ajh.26090
|
[2] |
梅琛, 佟红艳. 去甲基化药物治疗骨髓增生异常综合征的临床现状与前景[J]. 国际输血及血液学杂志, 2021, 44(4): 284-293. doi: 10.3760/cma.j.cn511693-20200327-0007
|
[3] |
佟红艳. 骨髓增生异常综合征去甲基化治疗: 地西他滨还是阿扎胞苷?[J]. 临床血液学杂志, 2019, 32(11): 841-846, 850. https://www.cnki.com.cn/Article/CJFDTOTAL-LCXZ201911006.htm
|
[4] |
吴雪, 陈宝安. 2014 NCCN指南骨髓增生异常综合征新进展[J]. 白血病·淋巴瘤, 2014, 23(9): 515-517. doi: 10.3760/cma.j.issn.1009-9921.2014.09.002
|
[5] |
万崇华, 陈明清, 张灿珍, 等. 癌症患者生命质量测定量表EORTC QLQ-C30中文版评介[J]. 实用肿瘤杂志, 2005, 20(4): 353-355. doi: 10.3969/j.issn.1001-1692.2005.04.028
|
[6] |
王佩, 董照, 原现华, 等. 阿扎胞苷对中高危骨髓增生异常综合征的临床疗效观察: 评《骨髓增生异常综合征肖志坚2018观点》[J]. 中国临床药理学与治疗学, 2021, 26(7): 841-841. https://www.cnki.com.cn/Article/CJFDTOTAL-YLZL202107021.htm
|
[7] |
CEDENA M T, RAPADO I, SANTOS-LOZANO A, et al. Mutations in the DNA methylation pathway and number of driver mutations predict response to azacitidine in myelodysplastic syndromes[J]. Oncotarget, 2017, 8(63): 106948-106961. doi: 10.18632/oncotarget.22157
|
[8] |
LIN M E, HOU H A, TSAI C H, et al. Dynamics of DNMT3A mutation and prognostic relevance in patients with primary myelodysplastic syndrome[J]. Clin Epigenetics, 2018, 10: 42. doi: 10.1186/s13148-018-0476-1
|
[9] |
陈园园, 石锐, 郭素青, 等. 地西他滨治疗伴DNA甲基转移酶基因突变的骨髓增生异常综合征患者的疗效分析[J]. 中国实验血液学杂志, 2020, 28(4): 1292-1297. https://www.cnki.com.cn/Article/CJFDTOTAL-XYSY202004040.htm
|
[10] |
BAO Z H, ZHAO H G, YU H E. Clinical efficacy and prognostic factors of decitabine for treatment of myelodysplastic syndrome[J]. Zhongguo Shi Yan Xue Ye Xue Za Zhi, 2018, 26(6): 1702-1707.
|
[11] |
FANG K, QI J Q, ZHOU M, et al. Clinical characteristics, prognosis, and treatment strategies of TP53 mutations in myelodysplastic syndromes[J]. Clin Lymphoma Myeloma Leuk, 2022, 22(4): 224-235. doi: 10.1016/j.clml.2021.09.013
|
[12] |
童春, 叶芳, 李宁宁, 等. 不同剂量地西他滨治疗高危骨髓增生异常综合征患者疗效及安全性的回顾性分析[J]. 中国实验血液学杂志, 2021, 29(6): 1845-1850. https://www.cnki.com.cn/Article/CJFDTOTAL-XYSY202106028.htm
|
[13] |
王欢, 郝建萍. 阿扎胞苷治疗对骨髓增生异常综合征患者近期疗效及生存率的影响[J]. 医学食疗与健康, 2022, 20(11): 4-6, 85. https://www.cnki.com.cn/Article/CJFDTOTAL-YXSL202211002.htm
|
[14] |
鲍振华, 赵洪国, 于虹娥. 地西他滨治疗骨髓增生异常综合征的临床观察及预后因素分析[J]. 中国实验血液学杂志, 2018, 26(6): 1702-1707. https://www.cnki.com.cn/Article/CJFDTOTAL-XYSY201806023.htm
|
[15] |
常炳庆, 孙婉玲. 去甲基化药物在骨髓增生异常综合征治疗中的进展[J]. 中国药物警戒, 2018, 15(9): 560-563. doi: 10.3969/j.issn.1672-8629.2018.09.012
|
[16] |
GARCIA-MANERO G, GRIFFITHS E A, STEENSMA D P, et al. Oral cedazuridine/decitabine for MDS and CMML: a phase 2 pharmacokinetic/pharmacodynamic randomized crossover study[J]. Blood, 2020, 136(6): 674-683. doi: 10.1182/blood.2019004143
|
[17] |
FENAUX P, SANTINI V, SPIRITI M A A, et al. A phase 3 randomized, placebo-controlled study assessing the efficacy and safety of epoetin-α in anemic patients with low-risk MDS[J]. Leukemia, 2018, 32(12): 2648-2658. doi: 10.1038/s41375-018-0118-9
|
[18] |
STAHL M, DEVEAUX M, WITTE T D, et al. The use of immunosuppressive therapy in MDS: clinical outcomes and their predictors in a large international patient cohort[J]. Blood Adv, 2018, 2(14): 1765-1772. doi: 10.1182/bloodadvances.2018019414
|
[19] |
MOSSNER M, JANN J C, NOWAK D, et al. Prevalence, clonal dynamics and clinical impact of TP53 mutations in patients with myelodysplastic syndrome with isolated deletion (5q) treated with lenalidomide: results from a prospective multicenter study of the German MDS study group (GMDS)[J]. Leukemia, 2016, 30(9): 1956-1959. doi: 10.1038/leu.2016.111
|
[20] |
ZHOU L, MCMAHON C, BHAGAT T, et al. Reduced SMAD7 leads to overactivation of TGF-beta signaling in MDS that can be reversed by a specific inhibitor of TGF-beta receptor I kinase[J]. Cancer Res, 2011, 71(3): 955-963. doi: 10.1158/0008-5472.CAN-10-2933
|
[21] |
WEI Y, DIMICOLI S, BUESO-RAMOS C, et al. Toll-like receptor alterations in myelodysplastic syndrome[J]. Leukemia, 2013, 27(9): 1832-1840. doi: 10.1038/leu.2013.180
|
[22] |
CARNEIRO B A, EL-DEIRY W S. Targeting apoptosis in cancer therapy[J]. Nat Rev Clin Oncol, 2020, 17(7): 395-417. doi: 10.1038/s41571-020-0341-y
|
[23] |
NAKADA D. Venetolax with azacitidine drains fuel from AML stem cells[J]. Cell Stem Cell, 2019, 24(1): 7-8. doi: 10.1016/j.stem.2018.12.005
|
[24] |
BEWERSDORF J P, ZEIDAN A M. Transforming growth factor (TGF)-β pathway as a therapeutic target in lower risk myelodysplastic syndromes[J]. Leukemia, 2019, 33(6): 1303-1312. doi: 10.1038/s41375-019-0448-2
|
[25] |
李瑞萍, 马艳萍. 罗特西普治疗骨髓增生异常综合征的研究进展[J]. 中国肿瘤临床, 2023, 50(18): 964-968. doi: 10.12354/j.issn.1000-8179.2023.20230874
|