期刊
BRITISH JOURNAL OF HAEMATOLOGY
卷 196, 期 4, 页码 969-974出版社
WILEY
DOI: 10.1111/bjh.17893
关键词
myelodysplastic syndrome; VEXAS syndrome; azacitidine
类别
Azacitidine may be effective in MDS associated with inflammatory/autoimmune diseases. A study on 116 VEXAS patients in France found that azacitidine treatment achieved clinical response in 46% of patients, suggesting its potential effectiveness in selected VEXAS patients with MDS.
Azacitidine can be effective in myelodysplastic syndromes (MDS) associated with inflammatory/autoimmune diseases. Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic syndrome (VEXAS) is a new monogenic autoinflammatory syndrome caused by somatic ubiquitin-like modifier-activating enzyme 1 (UBA1) mutation, often associated with MDS, whose treatment is difficult and not yet codified. Based on a French nationwide registry of 116 patients with VEXAS, we report the efficacy and safety of azacitidine treatment in 11 patients with VEXAS with MDS. Clinical response of VEXAS to azacitidine was achieved in five patients (46%), during 6, 8+, 12, 21, 27+ months respectively, suggesting that azacitidine can be effective in selected patients with VEXAS and associated MDS.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据