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.
Is immunotherapy safe and effective in patients with VEXAS syndrome? / Marvisi, C.; Muratore, F.; Cusenza, V. Y.; Nicoli, D.; Farnetti, E.; Bonanno, O.; Spaggiari, L.; Banzi, M.; Longo, C.; Salvarani, C.. - In: JOURNAL FOR IMMUNOTHERAPY OF CANCER. - ISSN 2051-1426. - 13:7(2025), pp. N/A-N/A. [10.1136/jitc-2025-012410]
Is immunotherapy safe and effective in patients with VEXAS syndrome?
Marvisi C.;Muratore F.;Bonanno O.;Spaggiari L.;Longo C.;Salvarani C.
2025
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.
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