Efficacy and Safety of Venetoclax in Combination with Hypometh-ylating Agents for the Treatment of High-Risk Myelodysplastic Syndromes
10.19746/j.cnki.issn1009-2137.2025.01.024
- VernacularTitle:维奈克拉联合去甲基化药物治疗高危组骨髓增生异常综合征患者的疗效及安全性分析
- Author:
Yang XU
1
;
Jian ZHANG
;
Zhi-Hong LIN
;
Jun CHEN
;
Li-Min LIU
;
Hui-Ying QIU
;
De-Pei WU
Author Information
1. 苏州大学附属第一医院血液科,江苏省血液研究所,国家血液系统疾病临床医学研究中心,江苏苏州 215006
- Publication Type:Journal Article
- Keywords:
myelodysplastic syndromes;
venetoclax;
hypomethylating agents;
adverse events
- From:
Journal of Experimental Hematology
2025;33(1):168-174
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To investigate the clinical efficacy and safety of venetoclax(VEN)in combination with hypomethylating agent(HMA)in the treatment of patients with high-risk myelodysplastic syndromes(MDS).Methods:A total of 30 patients with high-risk MDS who received the combination of VEN and HMA from March 2019 to November 2022 were included.The overall response rate(ORR),modified overall response rate(mORR),overall survival(OS),progression-free survival(PFS),and adverse events of all included patients were evaluated.Results:Among the 30 high-risk MDS patients treated with VEN combined with HMA regimen,24 cases achieved complete response(CR)/marrow complete response(mCR),2 cases achieved partial response(PR),the ORR was 24/30,the median OS was 28.1 months,and the median PFS was 28.1 months.In addition,patients who achieved complete remission/marrow complete remission after treatment had a significantly longer OS than those who did not.Moreover,12 patients were treated with allogeneic hematopoietic stem cell transplantation(allo-HSCT).There were grade 3 or higher hematologic adverse events including thrombocytopenia(14 cases),neutropenia(14 cases),febrile neutropenia(10 cases)and anemia(7 cases)as well as gastrointestinal adverse events of any grade,such as vomiting(7 cases),diarrhea(5 cases),and constipation(4 cases).Conclusion:VEN in combination with HMA is an effective and safe treatment option in patients with high-risk MDS.This regimen combined with allo-HSCT can improve the prognosis of these patients.Continuous attention to the monitoring and management of adverse events is essential for the patients'safety in this combination therapy.
