Blood · 2022 · 207 citations · 6 references
MedicinePharmacologyPharmacotherapyClinical PharmacologyTyrosine Kinase InhibitorsJak Inhibitor TherapyJak InhibitorsJak-stat Signaling PathwayJanus Kinase Inhibitor TherapyMulticenter TrialsTyrosine Kinase Inhibitor TherapyVexas Syndrome
VEXAS syndrome (vacuoles in myeloid progenitors, E1 ubiquitin activating enzyme, X-linked, autoinflammatory manifestations and somatic) is an autoinflammatory condition caused by somatically acquired UBA1 mutations. Heiblig et al report on an international retrospective analysis of 30 patients with VEXAS syndrome treated with different Janus kinase (JAK) inhibitors, finding encouraging evidence supporting the use of the JAK1/2 inhibitor ruxolitinib with clinical remissions and reductions in steroid use seen in the majority of patients.
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Sophie Georgin‐Lavialle, Benjamin Terrier, Alexis F. Guédon et al. · British Journal of Dermatology · 2021 · 417 citations · Full text
Medicine, Hematology, Pathology +16
Thibault Comont, Maël Heiblig, Étienne Rivière et al. · British Journal of Haematology · 2021 · 163 citations · Full text