Article
Successful azacitidine therapy for myelodysplastic syndrome associated with VEXAS syndrome.
International journal of hematology - 1 Jun 2023
Kataoka Asami, Mizumoto Chisaki, Kanda Junya, Iwasaki Makoto, Sakurada Maki, Oka Tomomi, Fujimoto Masakazu, Yamamoto Yosuke, Yamashita Kohei, Nannya Yasuhito, Ogawa Seishi, Takaori-Kondo Akifumi
Abstract excerpt
VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is caused by UBA1 somatic mutations and is characterized by late-onset systemic autoimmune inflammation and blood abnormalities such as cytopenia, vacuolation of myeloid/erythroblastic cells, and myelodysplastic syndrome (MDS). It is often resistant to immunosuppressive therapy, and no treatment strategy has been established. A 65-year-old...
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