病例报告:对阿扎胞苷治疗反应极佳的 VEXAS 综合征。

IF 3 3区 医学 Q2 HEMATOLOGY Annals of Hematology Pub Date : 2024-11-16 DOI:10.1007/s00277-024-06072-5
Tzvika Porges, Elli Rosenberg, Ofir Wolach, Iftach Sagy, Yehonatan Sherf, Itai Levi
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引用次数: 0

摘要

空泡、E1 酶、X 连锁、自身炎症、体细胞(VEXAS)综合征是一种由体细胞 UBA1 变异引起的炎症性疾病,其特征是晚发性全身自身免疫性炎症和血液异常。糖皮质激素能有效改善症状。然而,其他治疗方案的疗效有限,且效果短暂。在此,我们描述了一例患有 VEXAS 综合征的 69 岁男性患者,他的皮肤、肺部和血液系统均受累。他曾接受糖皮质激素治疗,在抗 IL-1 治疗失败后,他开始接受阿扎胞苷治疗,并获得了良好的血液学和临床反应。阿扎胞苷可能是治疗VEXAS综合征的一个合适选择,特别是由于炎症症状与阿扎胞苷反应之间的关系。
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Case report: VEXAS syndrome with excellent response to treatment with azacitidine.

Vacuoles, E1 enzyme, X-linked, auto inflammatory, somatic (VEXAS) syndrome is an inflammatory disorder caused by somatic UBA1 variants and is characterized by late-onset systemic autoimmune inflammation and blood abnormalities. Glucocorticoids ameliorate symptoms effectively. However, other treatment options have limited efficacy and a transient effect. Herein, we describe a case of a 69-year-old male patient with VEXAS syndrome with skin, lung and hematologic involvement. He was treated with glucocorticoids and after the failure with anti IL-1 he began treatment with azacitidine with excellent hematological and clinical response. Azacitidine may be a suitable option for treating VEXAS syndrome, especially due to the relationship between inflammatory symptoms and response to azacitidine.

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来源期刊
Annals of Hematology
Annals of Hematology 医学-血液学
CiteScore
5.60
自引率
2.90%
发文量
304
审稿时长
2 months
期刊介绍: Annals of Hematology covers the whole spectrum of clinical and experimental hematology, hemostaseology, blood transfusion, and related aspects of medical oncology, including diagnosis and treatment of leukemias, lymphatic neoplasias and solid tumors, and transplantation of hematopoietic stem cells. Coverage includes general aspects of oncology, molecular biology and immunology as pertinent to problems of human blood disease. The journal is associated with the German Society for Hematology and Medical Oncology, and the Austrian Society for Hematology and Oncology.
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