Clinical case demonstrates successful use of cemiplimab in controlling symptoms and UBA1 clones in VEXAS syndrome, indicating potential effectiveness despite safety concerns.
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.
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Marvisi et al. (2025) studied this question.
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