Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, and Somatic (VEXAS) Syndrome: A Case Report

空泡、E1酶、X连锁、自身炎症和躯体(VEXAS)综合征:病例报告

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Abstract

VEXAS is an acronym that stands for the technical terms of key descriptors of the condition: vacuoles, E1 enzyme, X-linked, autoinflammatory, and somatic (VEXAS) syndrome, which is a recently identified autoinflammatory disorder primarily affecting men older than 50 years of age. It is commonly associated with a somatic mutation in the X-linked ubiquitin-activating enzyme-encoding UBA1 gene. This condition manifests in a range of hematologic and systemic inflammatory symptoms, such as cytopenias, recurrent fevers, and an elevated risk for hematologic malignancies like myelodysplastic syndrome (MDS). Currently, glucocorticoids are the mainstay of treatment; however, biological therapies like janus kinase (JAK) and interleukin (IL) inhibitors have had varying degrees of effectiveness; unfortunately, despite these treatments, symptoms frequently persist and reoccur. Allogenic bone marrow transplantation is currently being considered as a possible treatment. This is a case of a 76-year-old male who presented with an unusual presentation of VEXAS syndrome and one who temporarily responded to treatment with doxycycline, an antibiotic with well-known anti-inflammatory qualities. This case report highlights our patient's unique presentation of VEXAS syndrome and emphasizes the importance of multiple bone marrow samples when the diagnosis is unclear. In this case, the results of the initial biopsy were inconclusive; however, following repeat bone marrow biopsy and the diagnosis of MDS, VEXAS syndrome was confirmed with testing for UBA1 mutations. This case underscores the importance of early and routine UBA1 mutation testing to facilitate timely diagnosis, improve patient outcomes, and guide the development of more effective treatment strategies. It also contributes to a broader understanding of VEXAS syndrome and its potential therapeutic approaches within the medical community.

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