The Journal of investigative dermatology · 2024
Topical Ruxolitinib in the Treatment of Necrobiosis Lipoidica: A Prospective, Open-Label Study.
СтатьяКлиническое исследование II фазыНазвание и аннотация: Перевод в очереди
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Hwang Angelina S, Kechter Jacob A, Li Xing, Hughes Alysia, Severson Kevin J, Boudreaux Blake, Bhullar Puneet, Nassir Shams, Yousif Miranda, Zhang Nan, Butterfield Richard J, Nelson Steven, Xing Xianying, Tsoi Lam C, Zunich Samantha, Sekulic Aleksandar, Pittelkow Mark, Gudjonsson Johann E, Mangold Aaron
Аннотация
Necrobiosis lipoidica (NL) is a rare granulomatous disease. There are few effective treatments for NL. We sought to investigate the efficacy and safety of the Jak1/2 inhibitor, ruxolitnib, in the treatment of NL and identify the biomarkers associated with the disease and treatment response. We conducted an open-label, phase 2 study of ruxolitinib in 12 patients with NL. We performed transcriptomic analysis of tissue samples before and after treatment. At week 12, the mean NL lesion score decreased by 58.2% (SD = 28.7%, P = .003). Transcriptomic analysis demonstrated enrichment of type I and type II IFN pathways in baseline disease. Weighted gene coexpression network analysis demonstrated post-treatment changes in IFN pathways with key hub genes IFNG and signal transducer and activator of transcription 1 gene STAT1. Limitations include small sample size and a study group limited to patients with <10% body surface area. In conclusion, ruxolitinib is an effective treatment for NL and targets the key pathogenic mediators of the disease.