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Case Reports
. 2025 Jul 31;13(7):e012410.
doi: 10.1136/jitc-2025-012410.

Is immunotherapy safe and effective in patients with VEXAS syndrome?

Chiara Marvisi  1   2 , Francesco Muratore  1   2 , Vincenza Ylenia Cusenza  1 , Davide Nicoli  1 , Enrico Farnetti  1 , Orsola Bonanno  1 , Lucia Spaggiari  1 , Maria Banzi  1 , Caterina Longo  1   2 , Carlo Salvarani  3   2
Affiliations
Case Reports

Is immunotherapy safe and effective in patients with VEXAS syndrome?

Chiara Marvisi et al. J Immunother Cancer. .

Abstract

The use of immune checkpoint inhibitors in VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is unknown. Concerns are particularly about their safety, due to their potential capacity to exacerbate inflammatory symptoms. Further, there is a lack of data about their role in controlling the UBA1 clones. In this clinical case, we report a successful use of cemiplimab in a patient with VEXAS syndrome and a concomitant diagnosis of metastatic squamous cell carcinoma.

Keywords: Immunotherapy.

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Conflict of interest statement

Competing interests: No, there are no competing interests.

Figures

Figure 1
Figure 1. (A) Timeline of the clinical history of the patient. (B and D) CT showing squamous cell carcinoma before (white and black asterisk) and after (C and E) treatment with cemiplimab. MDS, myelodysplastic syndrome; PDN, prednisone; SCC, squamous cell carcinoma; VAF, variant allele frequency.
See this image and copyright information in PMC

References

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