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Approaches for the diagnosis and treatment of VEXAS syndrome: the importance of clinical suspicion and the use of methotrexate

  • Maria De Santis,
  • Antonio Tonutti,
  • Francesca Motta,
  • Gabriele Todisco,
  • Nicla Manes,
  • Chiara Milanesi,
  • Rossella Caselli,
  • Serena Albertazzi,
  • Arturo Bonometti,
  • Carlo Selmi,
  • Matteo Giovanni Della Porta

摘要

Vacuoles, E1-enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) syndrome is caused by mutations in the UBA1 gene in myeloid precursors, leading to systemic inflammatory manifestations. We present the case of a 75-year-old man presenting with fever, panniculitis, and macrocytic anemia testing repeatedly negative for UBA1 mutations in peripheral blood samples, but ultimately found positive on bone marrow mononuclear cell DNA. The man has been successfully treated with prednisone and methotrexate.