Research progress of luspatercept in the treatment of SF3B1-mutated myelodysplastic syndrome
10.12354/j.issn.1000-8179.2024.20240680
- VernacularTitle:罗特西普在SF3B1突变骨髓增生异常综合征治疗中的研究进展
- Author:
Li JIAJING
1
;
Yu XIAODA
;
Wang ANAN
;
Guo JIANGANG
;
Liu BEI
Author Information
1. 兰州大学第一临床医学院(兰州市 730000)
- Keywords:
myelodysplastic syndrome(MDS);
SF3B1 mutation;
luspatercept;
efficacy;
safety
- From:
Chinese Journal of Clinical Oncology
2024;51(14):748-751
- CountryChina
- Language:Chinese
-
Abstract:
Myelodysplastic syndrome(MDS)is a heterogeneous myeloid tumor that originates from hematopoietic stem cells(HSCs)and is associated with a high risk of progression to acute myeloid leukemia(AML).Studies have shown that 90%of patients with MDS have gene mutations,of whom approximately 25%have SF3B1 mutations.In patients with MDS carrying this mutation,the TGF-β pathway is upregu-lated,inducing cell cycle arrest and thereby leading to erythroid ineffective hematopoiesis and pathological hematopoiesis.Luspatercept can be used as a ligand trap to capture TGF-β ligands,inhibit SMAD2/3 pathway activation,downregulate TGF-β pathway,and promote ad-vanced red blood cell maturation.Currently,it has been approved by the Food and Drug Administration(FDA)for the treatment of anemia in patients with low-risk MDS,and studies have shown that the response rate is higher in patients with SF3B1 mutations.This article will re-view the current status of luspatercept in the treatment of SF3B1 mutation-related MDS;it will also analyze its effectiveness and safety and provide therapeutic strategies for clinical use.
