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VEXAS syndrome - PubMed

4 hours ago
  • #Autoinflammatory disease
  • #VEXAS syndrome
  • #UBA1 mutation
  • VEXAS syndrome is a recently discovered disorder that connects hematology, immunology, and genetics.
  • It is caused by somatic mutations in the UBA1 gene in hematopoietic stem cells, disrupting protein homeostasis.
  • The condition mainly affects older men, presenting with inflammation, neutrophilic dermatoses, chondritis, and myelodysplastic features.
  • Diagnosis requires clinical signs and confirmation of UBA1 mutations.
  • Prognosis is often poor, and treatment includes glucocorticoids, cytokine blockade, hypomethylating agents, and allogeneic stem cell transplantation as a potential cure.