Case report: VEXAS syndrome: first documented cases in Latin America
Introduction VEXAS syndrome (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) is a recently identified disorder associated with somatic mutations in the UBA1 gene. Predominantly affecting adult males, it is characterized by a wide range of autoinflammatory symptoms and hematologic abnormalities. Methods We present three cases from Latin America, marking the first reported occurrences in this region, to illustrate the clinical variability and diagnostic challenges of VEXAS syndrome. Results Each patient exhibited unique clinical presentations, including refractory autoinflammatory symptoms, myelodysplastic syndrome, and bone marrow vacuolization. All cases were confirmed via genetic testing, revealing pathogenic UBA1 mutations alongside other genetic variants commonly linked with myeloid neoplasms. Discussion These findings underscore the importance of considering VEXAS syndrome in patients with unexplained inflammatory and hematologic symptoms. The coexistence of UBA1 mutations with other genetic variants suggests a potential overlap with clonal hematopoiesis, complicating the clinical picture. These cases contribute to the understanding of VEXAS syndrome and highlight the need for increased awareness and diagnostic testing in diverse populations to ensure early and accurate diagnosis.
Authors
- Carolina Ottati
- Matilde Boada (ORCID: https://orcid.org/0000-0003-3836-8751)
- Martín Yandián
- Diego Arango López (ORCID: https://orcid.org/0000-0001-5831-6073)
- Inés Gervaz
- Ana Inés Catalán (ORCID: https://orcid.org/0009-0003-2458-7272)
- Patricia Kutscher
- Gabriela Vidal‐Senmache
- Sofía Grille (ORCID: https://orcid.org/0000-0002-0590-7109)
- Percy Ortiz-Guerra (ORCID: https://orcid.org/0000-0003-4641-6127)
- Lilián Díaz (ORCID: https://orcid.org/0009-0006-6397-0129)
Institutions
- Universidad de la República de Uruguay (UY)
- Hospital Nacional Cayetano Heredia (PE)
- Hospital de Clínicas (UY)
- Centro Uruguayo de Imagenología Molecular (UY)
Publication Details
- Journal
- Frontiers in Hematology
- Published
- 2025-01-16
- DOI
- https://doi.org/10.3389/frhem.2024.1501337
- Primary Topic
- Otitis Media and Relapsing Polychondritis
- Type
- article
- Field-Weighted Citation Impact
- 0.00