Ruxolitinib Rescues Multiorgan Clinical Autoimmunity in Patients With APS-1

    December 2023 in “ Journal of clinical immunology
    Romain Lévy, A. Escudier, Paul Bastard, Coralie Briand, Laura Polivka, Athanasia Stoupa, Cécile Talbotec, Anya Rothenbühler, Marina Charbit, Dominique Debray, Christine Bodemer, Jean‐Laurent Casanova, Agnès Linglart, Bénédicte Neven
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    TLDR Ruxolitinib significantly improves multiple autoimmune conditions in APS-1 patients.
    The study on ruxolitinib, a JAK inhibitor, in three patients with autoimmune polyendocrine syndrome type-1 (APS-1) over at least 30 months showed significant clinical improvements in multiple autoimmune conditions, including alopecia, nail dystrophy, and mucosal candidiasis, among others. Hair regrowth was noted after 3 months, with full resolution of alopecia after 12 months. The treatment was well-tolerated with no major adverse events, except for transient anemia in one patient. These results suggest that JAK inhibitors like ruxolitinib could be effective for APS-1, but larger studies are needed to confirm these findings and evaluate long-term safety.
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