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Clinical Trial
. 2022 Nov;36(11):2739-2742.
doi: 10.1038/s41375-022-01698-8. Epub 2022 Sep 14.

A Phase II prospective trial of azacitidine in steroid-dependent or refractory systemic autoimmune/inflammatory disorders and VEXAS syndrome associated with MDS and CMML

Arsene Mekinian #  1 , Lin Pierre Zhao #  2 , Sylvie Chevret  3 , Kristell Desseaux  3 , Laurent Pascal  4 , Thibaut Comont  5 , Alexandre Maria  6 , Pierre Peterlin  7 , Louis Terriou  8 , Maud D'Aveni Piney  9 , Marie-Pierre Gourin  10 , Norbert Vey  11 , Odile Beyne Rauzy  5 , Vincent Grobost  12 , Holy Bezanahary  13 , Sophie Dimicoli-Salazar  14 , Anne Banos  15 , Stefan Wickenhauser  16 , Benoit De Renzis  17 , Eric Durot  18 , Shanti Natarajan-Amé  19 , Laurent Voillat  10 , Fatiha Chermat  2 , Karine Lemaire  2 , Vincent Jachiet  20 , Chantal Himberlin  18 , Sylvain Thépot  21 , Jose Miguel Torregrosa Diaz  22 , Laurent Frenzel  23 , Emmanuel Gyan  24 , Guillaume Denis  25 , Pierre Hirsch  26 , Olivier Kosmider  27 , Lionel Ades  2 , Olivier Fain  20 , Pierre Fenaux  2
Affiliations
Clinical Trial

A Phase II prospective trial of azacitidine in steroid-dependent or refractory systemic autoimmune/inflammatory disorders and VEXAS syndrome associated with MDS and CMML

Arsene Mekinian et al. Leukemia. 2022 Nov.
No abstract available

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References

    1. Mekinian A, Grignano E, Braun T, Decaux O, Liozon E, Costedoat-Chalumeau N, et al. Systemic inflammatory and autoimmune manifestations associated with myelodysplastic syndromes and chronic myelomonocytic leukaemia: a French multicentre retrospective study. Rheumatology. 2016;55:291–300. - DOI - PubMed
    1. Zhao L-P, Boy M, Azoulay C, Clappier E, Sébert M, Amable L, et al. Genomic landscape of MDS/CMML associated with systemic inflammatory and autoimmune disease. Leukemia. 2021;35:2720–4. - DOI - PubMed
    1. Beck DB, Ferrada MA, Sikora KA, Ombrello AK, Collins JC, Pei W, et al. Somatic mutations in UBA1 and severe adult-onset autoinflammatory disease. N Engl J Med. 2020;383:2628–38. - DOI - PubMed - PMC
    1. Zhao L-P, Schell B, Sébert M, Kim R, Lemaire P, Boy M, et al. Prevalence of UBA1 mutations in MDS/CMML patients with systemic inflammatory and auto-immune disease. Leukemia. 2021;35:2731–3. - DOI - PubMed
    1. Grayson PC, Patel BA, Young NS. VEXAS syndrome. Blood. 2021;137:3591–4. - PubMed - PMC

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