Options for patients with myelodysplastic syndromes after the treatment failure of hypomethylating agent
10.3760/cma.j.issn.1009-9921.2017.10.001
- VernacularTitle:骨髓增生异常综合征患者低甲基化治疗失败后的治疗选择
- Author:
Xudong TANG
1
;
Lu ZHANG
;
Xiupeng YANG
;
Yufeng TANG
;
Dexiu WANG
Author Information
1. 中国中医科学院西苑医院血液科
- Keywords:
Myelodysplastic syndromes;
Hypomethylating agent;
Treatment failure
- From:
Journal of Leukemia & Lymphoma
2017;26(10):577-581
- CountryChina
- Language:Chinese
-
Abstract:
The treatment of patients with myelodysplastic syndromes (MDS) has included primarily supportive care (blood transfusions, colony stimulating agents, iron chelation, etc.) and new drugs, including 5-azacytidine, deoxyazacytidine, and lenalidomide. For those who are no longer benefiting from these agents, there is nearly nothing to do with effective therapies. When those drugs are ineffective, what the doctors could do is optimizing the delivery of hypomethylating agents (HMT) by dosing appropriately, sequencing appropriately, and using thoughtful combinations; as well as improving drug formulations (oral formulations and/or novel formulations) and working toward better selection of patients for best upfront mutation-directed therapy. Hopefully, the drug targeting and patient selection for optimal HMT treatment of MDS will be improved. Furthermore, ongoing research is focused on identifying unique agents to rescue MDS patients who have progressed despite HMT. Agents such as rigosertib are now focused on its application in specific MDS populations who might most likely benefit from this therapeutic approach [primary refractory and high-risk international prognostic scoring system (IPSS) patients]. Doctors eagerly await results of single-agent programmed death 1 (PD-1) and its inhibitors, or combination with HMT to the upfront and relapsed MDS setting. For the minority of patients who have specific targetable mutations, the selective agents (IDH1/2) are highly promising. Bone marrow transplantation remains the only offer for cure, but is depressingly unrealistic given the majority of the elderly and frail patients at the time of MDS progression.
