Allogenic haematopoietic stem cell transplantation in VEXAS: A review of 33 patients

SB Ali, C Gurnari - Clinical Rheumatology, 2024 - Springer
Abstract Vacuolation, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome is
a multisystem disease due to a genetic mutation in the ubiquitin-activating enzyme (UBA1) …

Molecular and clinical presentation of UBA1-mutated myelodysplastic syndromes

M Sirenko, E Bernard, M Creignou, D Domenico… - Blood, 2024 - ashpublications.org
Mutations in UBA1, which are disease-defining for VEXAS (vacuoles, E1 enzyme, X-linked,
autoinflammatory, somatic) syndrome, have been reported in patients diagnosed with …

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

C Gurnari, L Koster, L Baaij, M Heiblig… - Blood …, 2024 - ashpublications.org
Somatic mutations of the X-linked UBA1 gene in myeloid progenitors constitute the
molecular underpinnings of VEXAS, typically occurring in men during the sixth decade of …

Latest Insights and Therapeutic Advances in Myelodysplastic Neoplasms

P Niscola, V Gianfelici, M Giovannini, D Piccioni… - Cancers, 2024 - mdpi.com
Simple Summary Several recent studies have demonstrated encouraging results in treating
patients with myelodysplastic syndromes/neoplasms (MDSs). This review focuses on the …

VEXAS syndrome: complete molecular remission after hypomethylating therapy

K Sockel, K Götze, C Ganster, M Bill, JA Georgi… - Annals of …, 2024 - Springer
The VEXAS syndrome, a genetically defined autoimmune disease, associated with various
hematological neoplasms has been attracting growing attention since its initial description in …

VEXAS syndrome: an adult-onset autoinflammatory disorder with underlying somatic mutation

I Kötter, M Krusche - Current Opinion in Rheumatology, 2025 - journals.lww.com
In the current review, we discuss the novelties for VEXAS, including pathogenic pathways,
epidemiological data, diagnostic criteria and algorithms, treatment options and …

[HTML][HTML] How Protein Ubiquitination Can Influence Cytokine Expression—Updated Review on Autoinflammatory VEXAS Syndrome

D Majer, M Kujawińska, P Limanówka, Ł Sędek - Immuno, 2024 - mdpi.com
VEXAS syndrome is a new disease entity with symptoms that can mimic hematological,
rheumatic and dermatological diseases. It is important to take a multidisciplinary approach to …

Hypomethylating agents for patients with VEXAS without myelodysplastic syndrome: Clinical outcome and longitudinal follow‐up of vacuolization and UBA1 clonal …

JR Álamo, LM Torres, S Castaño‐Díez… - British Journal of …, 2025 - Wiley Online Library
VEXAS syndrome is a haemato‐inflammatory disease caused by somatic UBA1 mutations
and characterized by cytoplasmic vacuoles in myeloid and erythroid precursor cells …

Novel use of Siltuximab in a patient with VEXAS Syndrome

B Cáceres-Nazario, J Rivenbark, MK Saha… - Annals of …, 2024 - Springer
VEXAS syndrome (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) is an
increasingly recognized disorder that occurs due to somatic mutations of a ubiquitin …

VEXAS syndrome: is it more a matter of inflammation or hematopoietic clonality? A case series approach to diagnosis, therapeutic strategies and transplant …

A Costa, F Pilo, M Pettinau, M Piga, P Carboni… - Annals of …, 2025 - Springer
VEXAS syndrome is a complex hemato-inflammatory disorder, driven by somatic mutations
in the UBA1 gene within hematopoietic precursor cells. It is characterized by systemic …