Risks of Ruxolitinib in STAT1 Gain-of-Function-Associated Severe Fungal Disease

Abstract

Heterozygous <i>STAT1</i> gain-of-function (GOF) mutations are associated with chronic mucocutaneous candidiasis and a broad spectrum of infectious, inflammatory, and vascular manifestations. We describe therapeutic failures with the Janus Kinase (JAK) inhibitor ruxolitinib in 2 <i>STAT1</i> GOF patients with severe invasive or cutaneous fungal infections.