1. Topical Ruxolitinib in the Treatment of Necrobiosis Lipoidica: A Prospective, Open-Label Study.
- Author
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Hwang AS, Kechter JA, Li X, Hughes A, Severson KJ, Boudreaux B, Bhullar P, Nassir S, Yousif M, Zhang N, Butterfield RJ, Nelson S, Xing X, Tsoi LC, Zunich S, Sekulic A, Pittelkow M, Gudjonsson JE, and Mangold A
- Subjects
- Humans, Female, Male, Prospective Studies, Middle Aged, Adult, Treatment Outcome, Aged, Gene Expression Profiling, Administration, Topical, Janus Kinase Inhibitors therapeutic use, Janus Kinase Inhibitors administration & dosage, Nitriles, Pyrimidines therapeutic use, Pyrazoles therapeutic use, Pyrazoles administration & dosage, Necrobiosis Lipoidica drug therapy, Necrobiosis Lipoidica genetics, Necrobiosis Lipoidica pathology
- Abstract
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., (Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved.)
- Published
- 2024
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