Abstract
We report the case of the youngest patient described with VEXAS syndrome associated with MDS-IB1, successfully treated with azacitidine-venetoclax and allogeneic stem cell transplant.
This is a preview of subscription content, log in via an institution to check access.
Similar content being viewed by others
Data availability
No datasets were generated or analysed during the current study.
References
Beck DB, Ferrada MA, Sikora KA, Ombrello AK, Collins JC, Pei W et al (2020) Somatic mutations in UBA1 and severe adult-onset Autoinflammatory Disease. N Engl J Med 383(27):2628–2638
Khoury JD, Solary E, Abla O, Akkari Y, Alaggio R, Apperley JF et al (2022) The 5th edition of the World Health Organization Classification of Haematolymphoid Tumours: myeloid and Histiocytic/Dendritic neoplasms. Leukemia 36(7):1703–1719
Bourbon E, Heiblig M, Gerfaud Valentin M, Barba T, Durel CA, Lega JC et al (2021) Therapeutic options in VEXAS syndrome: insights from a retrospective series. Blood 137(26):3682–3684
Heiblig M, Ferrada MA, Koster MT, Barba T, Gerfaud-Valentin M, Mékinian A et al (2022) Ruxolitinib is more effective than other JAK inhibitors to treat VEXAS syndrome: a retrospective multicenter study. Blood 140(8):927–931
Comont T, Kosmider O, Heiblig M, Terrier B, Bouscary D, Le Guenno G et al (2023) Azacitidine for patients with Vexas Syndrome: data from the French Vexas Registry. Blood 142(Supplement 1):4604–4604
Yohei Kirino K, Takase-Minegishi N, Tsuchida L, Hirahara Y, Kunishita R, Yoshimi et al (2021) Tocilizumab in VEXAS relapsing polychondritis: a single-center pilot study in Japan. Ann Rheum Dis 80(11):1501
Diarra A, Duployez N, Fournier E, Preudhomme C, Coiteux V, Magro L et al (2022) Successful allogeneic hematopoietic stem cell transplantation in patients with VEXAS syndrome: a 2-center experience. Blood Adv 6(3):998–1003
Gurnari C, Koster L, Baaij L, Heiblig M, Yakoub-Agha I, Collin M et al (2024) Allogeneic hematopoietic cell transplantation for VEXAS syndrome: results of a multicenter study of the EBMT. Blood Adv 8(6):1444–1448
Georgin-Lavialle S, Terrier B, Guedon AF, Heiblig M, Comont T, Lazaro E et al (2022) Further characterization of clinical and laboratory features in VEXAS syndrome: large‐scale analysis of a multicentre case series of 116 French patients. Br J Dermatol 186(3):564–574
Funding
The authors did not receive support from any organization for the submitted work.
Author information
Authors and Affiliations
Contributions
H.G. wrote the first draft. H.G. and J.O. performed and collected the biological data. M.K., A.A., J.S. and A.C-H. collected the clinical data. M.K, A.A and A.C-H. provided patient care. All authors reviewed the manuscript and approved the submitted version.
Corresponding author
Ethics declarations
Competing interests
The authors declare no competing interests.
Additional information
Publisher’s Note
Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
Rights and permissions
Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.
About this article
Cite this article
Guerineau, H., Kohn, M., Al Hamoud, A. et al. Could it be VEXAS?. Ann Hematol 103, 2169–2171 (2024). https://doi.org/10.1007/s00277-024-05750-8
Received:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1007/s00277-024-05750-8