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Multicenter Study
. 2024 Mar 26;8(6):1444-1448.
doi: 10.1182/bloodadvances.2023012478.

Allogeneic hematopoietic cell transplantation for VEXAS syndrome: results of a multicenter study of the EBMT

Carmelo Gurnari  1   2 , Linda Koster  3 , Laurien Baaij  3 , Mael Heiblig  4 , Ibrahim Yakoub-Agha  5 , Matthew Collin  6 , Jakob Passweg  7 , Claude E Bulabois  8 , Anjum Khan  9 , Michael Loschi  10 , Fabrizio Carnevale-Schianca  11 , Elena Crisà  11 , Daniela Caravelli  11 , Jürgen Kuball  12 , Francesco Saraceni  13 , Attilio Olivieri  13 , Alessandro Rambaldi  14 , Austin G Kulasekararaj  15 , Patrick J Hayden  16 , Manuela Badoglio  17 , Francesco Onida  18 , Christof Scheid  19 , Franco Franceschini  20 , Arsène Mekinian  21 , Sinisa Savic  22 , Maria Teresa Voso  1 , Joanna Drozd-Sokolowska  23 , John A Snowden  24 , Kavita Raj  25 , Tobias Alexander  26 , Marie Robin  27 , Raffaella Greco  28 , Donal P McLornan  25
Affiliations
Multicenter Study

Allogeneic hematopoietic cell transplantation for VEXAS syndrome: results of a multicenter study of the EBMT

Carmelo Gurnari et al. Blood Adv. .
No abstract available

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Conflict of interest statement

Conflict-of-interest disclosure: The authors declare no competing financial interests.

Figures

Figure 1.
Figure 1.
VEXAS features and post-transplant outcomes. (A) Bar graph shows the distribution of patients according to disease phenotypes and association with myeloid neoplasia (myelodysplastic syndrome, MDS or myeloproliferative neoplasia, MPN). (B) Pie chart detailing the frequency of the UBA1 mutations identified in our cohort. (C) Bar graph shows VEXAS clinical manifestations. (D) Pie chart showcases the MDS diagnosis according to the WHO 2016 MDS classification. (E) Kaplan-Meier curve shows the probability of OS in 19 patients with VEXAS undergoing allo-HCT. Numbers at risk are indicated below the curve. (F) Kaplan-Meier curve shows TRM in 19 patients with VEXAS undergoing allo-HCT. Numbers at risk are indicated below the curve.
See this image and copyright information in PMC

References

    1. Gurnari C, McLornan DP. Update on VEXAS and role of allogeneic bone marrow transplant: considerations on behalf of the Chronic Malignancies Working Party of the EBMT. Bone Marrow Transplant. 2022;57(11):1642–1648. - PubMed
    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. Gurnari C, Visconte V. From bone marrow failure syndromes to VEXAS: disentangling clonal hematopoiesis, immune system, and molecular drivers. Leuk Res. 2023;127 - PubMed
    1. Bruno A, Gurnari C, Alexander T, Snowden JA, Greco R, Autoimmune Diseases Working Party of the European Society for Blood and Marrow Transplantation Autoimmune manifestations in VEXAS: Opportunities for integration and pitfalls to interpretation. J Allergy Clin Immunol. 2023;151(5):1204–1214. - PubMed
    1. Loschi M, Roux C, Sudaka I, et al. Allogeneic stem cell transplantation as a curative therapeutic approach for VEXAS syndrome: a case report. Bone Marrow Transplant. 2022;57(2):315–318. - PubMed

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