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. 2025 Jun 1;110(6):1432-1435.
doi: 10.3324/haematol.2024.286641. Epub 2025 Jan 30.

Very long-term remission with azacitidine in VEXAS syndrome

Lin-Pierre Zhao  1 , Vincent Jachiet  2 , Olivier Kosmider  3 , Lise Larcher  4 , Emmanuelle Clappier  4 , Marie Sébert  5 , Lionel Adès  5 , Pierre Hirsch  6 , Thibault Mahévas  7 , Maxime Battistella  8 , Jean-David Bouaziz  7 , Arsène Mekinian  2 , Pierre Fenaux  5
Affiliations

Very long-term remission with azacitidine in VEXAS syndrome

Lin-Pierre Zhao et al. Haematologica. .
No abstract available

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Figures

Figure 1.
Figure 1.
Longitudinal monitoring of clinical course, hematologic parameters, and molecular burden in relation to treatment responses. Timeline showing the association of clonal burden (left axis), hematologic parameters and C-reactive protein (CRP) level (right axis), bone marrow (BM) findings, transfusion dependency and their relation to treatment. Clonal cell fractions (CCF) (representing the proportion of mutated cells) were assessed using next-generation sequencing (sensitivity threshold 1%). ANC: absolute neutrophil count; AZA: azacitidine; EPO: erythropoietin; Hb: hemoglobin; Plat: platelets; MDS-MLD: myelodysplastic syndrome with multilineage dysplasia (according to WHO 2016).
Figure 2.
Figure 2.
Multisystemic manifestations and histopathological features of VEXAS. (A) Erythematous papules and plaques of the upper right arm. (B) Isolated chondritis of the left ear. (C) Histopathological features of skin biopsy in 2020 comprise a superficial and deep dermal perivascular infiltrate (upper left, HES, x30 magnification) made of lymphocytes, mononucleated histiocytoid cells with incompletely segmented nuclei, few mature neutrophils and eosinophils (lower left, HES, x400 magnification). Histiocytoid cells express CD163 (upper right, x400 magnification) and myeloperoxidase (lower right, x400 magnification), indicating immature non-blastic myeloid cells.
See this image and copyright information in PMC

References

    1. Beck DB, Ferrada MA, Sikora KA, et al. . Somatic mutations in UBA1 and severe adult-onset autoinflammatory disease. N Engl J Med. 2020;383(27):2628-2638. - PMC - PubMed
    1. Diarra A, Duployez N, Fournier E, et al. . Successful allogeneic hematopoietic stem cell transplantation in patients with VEXAS syndrome: a 2-center experience. Blood Adv. 2022;6(3):998-1003. - PMC - PubMed
    1. Arber DA, Orazi A, Hasserjian R, et al. . The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016;127(20):2391-2405. - PubMed
    1. Cheson BD, Greenberg PL, Bennett JM, et al. . Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. Blood. 2006;108(2):419-425. - PubMed
    1. Raaijmakers MHGP, Hermans M, Aalbers A, et al. . Azacytidine treatment for VEXAS syndrome. Hemasphere. 2021;5(12):e661. - PMC - PubMed

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