Azacitidine for patients with Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic syndrome (VEXAS) and myelodysplastic syndrome : data from the French VEXAS registry
© 2021 British Society for Haematology and John Wiley & Sons Ltd..
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.
| Errataetall: |
CommentIn: Br J Haematol. 2022 Feb;196(4):804-805. - PMID 34729765 |
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| Medienart: |
E-Artikel |
| Erscheinungsjahr: |
2022 |
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| Erschienen: |
2022 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:196 |
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| Enthalten in: |
British journal of haematology - 196(2022), 4 vom: 14. Feb., Seite 969-974 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Comont, Thibault [VerfasserIn] |
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| Links: |
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| Themen: |
Antimetabolites, Antineoplastic |
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| Anmerkungen: |
Date Completed 01.03.2022 Date Revised 01.03.2022 published: Print-Electronic CommentIn: Br J Haematol. 2022 Feb;196(4):804-805. - PMID 34729765 Citation Status MEDLINE |
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| doi: |
10.1111/bjh.17893 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM331920204 |
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| 520 | |a 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 | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a VEXAS syndrome | |
| 650 | 4 | |a azacitidine | |
| 650 | 4 | |a myelodysplastic syndrome | |
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| 650 | 7 | |a Azacitidine |2 NLM | |
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| 700 | 1 | |a Terriou, Louis |e verfasserin |4 aut | |
| 700 | 1 | |a Rossignol, Julien |e verfasserin |4 aut | |
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