Is immunotherapy safe and effective in patients with VEXAS syndrome?

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 con...

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Bibliographic Details
Main Authors: Chiara Marvisi, Francesco Muratore, Carlo Salvarani, Davide Nicoli, Enrico Farnetti, Lucia Spaggiari, Vincenza Ylenia Cusenza, Orsola Bonanno, Maria Banzi, Caterina Longo
Format: Article
Language:English
Published: BMJ Publishing Group 2025-07-01
Series:Journal for ImmunoTherapy of Cancer
Online Access:https://jitc.bmj.com/content/13/7/e012410.full
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Summary: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.
ISSN:2051-1426