Efficacy and Safety of Venetoclax in Combination with Hypomethylating Agents for the Treatment of High-Risk Myelodysplastic Syndromes.
10.19746/j.cnki.issn.1009-2137.2025.01.024
- Author:
Yang XU
1
;
Jian ZHANG
1
;
Zhi-Hong LIN
2
;
Jun CHEN
2
;
Li-Min LIU
3
;
Hui-Ying QIU
1
;
De-Pei WU
1
Author Information
1. Department of Hematology, The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology, National Clinical Research Center for Hematologic Diseases, Suzhou 215006, Jiangsu Province, China.
2. Department of Hematology, Suzhou Yongding Hospital, Suzhou 215299, Jiangsu Province, China.
3. Department of Hematology, Soochow Hopes Hematology Hospital, Suzhou 215128, Jiangsu Province, China.
- Publication Type:Journal Article
- Keywords:
myelodysplastic syndromes;
venetoclax;
hypomethylating agents;
adverse events
- MeSH:
Humans;
Myelodysplastic Syndromes/drug therapy*;
Sulfonamides/therapeutic use*;
Bridged Bicyclo Compounds, Heterocyclic/therapeutic use*;
Female;
Male;
Treatment Outcome;
Middle Aged;
Aged;
Antineoplastic Combined Chemotherapy Protocols/therapeutic use*;
Adult
- 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.
