Article

Primary CD34 <sup>+</sup> cells of patients with VEXAS syndrome are highly sensitive to targeted treatment with TAK-243 and pevonedistat

2025-05-24

Abstract excerpt

Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) syndrome is an autoinflammatory disease characterized by somatically acquired UBA1 mutations in hematopoietic stem cells. VEXAS patients present with significant therapeutic challenges such as severe treatment-resistant inflammation and predisposition to myeloid malignancies. In this study, we evaluated the efficacy of TAK-243, a UBA1 inhibitor, and...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 37abce78-0fd6-5774-bbd5-2bac0564a8cb
DOI: 10.1101/2025.05.22.653244

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Primary CD34 <sup>+</sup> cells of patients with VEXAS syndrome are highly sensitive to targeted treatment with TAK-243 and pevonedistatDOI 10.1101/2025.05.22.653244Read

VEXAS syndrome.Semantic proximity Read

A clinical, histopathological, and molecular study of two cases of VEXAS syndrome without a definitive myeloid neoplasm.Semantic proximity Read

Looking beyond VEXAS: Coexistence of undifferentiated systemic autoinflammatory disease and myelodysplastic syndrome.Semantic proximity Read

Clonal hematopoiesis meets an autoinflammatory disease: the new paradigm of VEXAS syndrome.Semantic proximity Read

VEXAS: A review of current understandings and emerging treatment strategies.Semantic proximity Read

An update on VEXAS syndrome.Semantic proximity Read

Benign and malignant hematologic manifestations in patients with VEXAS syndrome due to somatic mutations in UBA1.Semantic proximity Read

VEXAS syndrome: a comprehensive review of pathogenesis, clinical spectrum, and therapeutic strategies.Semantic proximity Read

VEXAS syndrome: Clinical, hematologic features and a practical approach to diagnosis and management.Semantic proximity Read

American College of Rheumatology Guidance Statement for Diagnosis and Management of VEXAS Developed by the International VEXAS Working Group Expert Panel.Semantic proximity Read

VEXAS syndrome in adults: A narrative review of genomic pathogenesis, multisystem phenotypes, diagnostic algorithms, and emerging therapeutic strategies.Semantic proximity Read

Somatic mutations in VEXAS Syndrome and Erdheim-Chester disease: Inflammatory myeloid diseases.Semantic proximity Read