AI Article Synopsis
- * A study using data from a French nationwide registry included 116 VEXAS patients, focusing on 11 individuals with concurrent MDS treated with azacitidine.
- * Out of the 11 treated patients, five (46%) exhibited clinical responses to azacitidine, highlighting its potential as a treatment option for select VEXAS patients experiencing MDS.
Article Abstract
Azacitidine can be effective in myelodysplastic syndromes (MDS) associated with inflammatory/autoimmune diseases. Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic syndrome (VEXAS) is a new monogenic autoinflammatory syndrome caused by somatic ubiquitin-like modifier-activating enzyme 1 (UBA1) mutation, often associated with MDS, whose treatment is difficult and not yet codified. Based on a French nationwide registry of 116 patients with VEXAS, we report the efficacy and safety of azacitidine treatment in 11 patients with VEXAS with MDS. Clinical response of VEXAS to azacitidine was achieved in five patients (46%), during 6, 8+, 12, 21, 27+ months respectively, suggesting that azacitidine can be effective in selected patients with VEXAS and associated MDS.
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| http://dx.doi.org/10.1111/bjh.17893 | DOI Listing |
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Article Synopsis
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