Utility of Ruxolitinib in a Child with Chronic Mucocutaneous Candidiasis Caused by a Novel STAT1 Gain-of-Function Mutation.
Markéta BloomfieldVeronika KanderováZuzana ParačkováPetra VrabcováMichael SvatoňEva FroňkováMartina FejtkováRadana ZachováMichal RatajIrena ZentsováTomáš MilotaAdam KlocperkTomáš KalinaAnna ŠediváPublished in: Journal of clinical immunology (2018)
JAK1/2 inhibition with ruxolitinib represents a viable option for treatment of refractory CMC, if HSCT is not considered. However, long-term administration is necessary, as the effect is not sustained after treatment discontinuation.