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Review
. 2024 Nov 19;13(22):6970.
doi: 10.3390/jcm13226970.

VEXAS Syndrome: A Comprehensive Review of Current Therapeutic Strategies and Emerging Treatments

Safi Alqatari  1 , Abdulaziz A Alqunais  2 , Shahad M Alali  2 , Mohammed A Alharbi  2 , Manal Hasan  1 , Mohammed D Al Shubbar  2
Affiliations
Review

VEXAS Syndrome: A Comprehensive Review of Current Therapeutic Strategies and Emerging Treatments

Safi Alqatari et al. J Clin Med. .

Abstract

VEXAS syndrome is a recently identified autoinflammatory disorder resulting from somatic mutations in the UBA1 gene, leading to a complex spectrum of severe inflammatory and hematologic manifestations. The absence of established treatment guidelines and the variability in clinical presentation make its management particularly challenging. Current therapeutic approaches are often based on limited evidence, and their effectiveness remains inconsistent. This review seeks to consolidate the existing knowledge on therapeutic strategies for VEXAS syndrome, offering a critical evaluation of their efficacy and addressing the gaps in the current literature. As the clinical recognition of VEXAS grows, there is an urgent need to explore more targeted, effective treatments that can address both the inflammatory and hematologic aspects of the disease. By providing a comprehensive analysis of the current therapeutic landscape, this review aims to guide clinicians and researchers toward developing more effective, long-term management strategies for this life-threatening condition.

Keywords: UBA1 mutation; VEXAS syndrome; autoinflammatory disorder; hematological disorders.

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Conflict of interest statement

The authors declare no conflicts of interest.

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