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A child with severe juvenile dermatomyositis treated with ruxolitinib

Abstract : Sir, We read with interest the article by Ladislau et al. (2018), highlighting the key role of type I interferon (IFN) in the pathophysiology of the muscle disease observed in dermatomyositis and reporting the short-term efficacy of ruxolitinib in four adult patients. Type I IFNs bind a unique common receptor (IFNAR) and subsequently activate the Janus Kinase 1 (JAK1) and tyrosine kinase 2, which, in turn, leads to the phosphorylation of signal transducer of activator of transcription (STAT) 1 and STAT2 driving the expression of IFN-stimulated genes (ISGs). Here, we describe the use of the JAK1/2 selective inhibitor ruxolitinib in a child with severe vasculopathic refractory juvenile dermatomyositis (JDM). Informed parental consent was obtained for the use of ruxolitinib on a compassionate basis and the associated experimental work, conducted in compliance with the 1975 Declaration of Helsinki [...]
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Contributor : Marie-Christine Vougny Connect in order to contact the contributor
Submitted on : Friday, December 21, 2018 - 9:36:26 AM
Last modification on : Thursday, October 20, 2022 - 3:10:07 PM

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Florence Aeschlimann, Marie-Louise Frémond, Darragh Duffy, Gillian Rice, Jean-Luc Charuel, et al.. A child with severe juvenile dermatomyositis treated with ruxolitinib. Brain - A Journal of Neurology , 2018, 141 (11), pp.e80. ⟨10.1093/brain/awy255⟩. ⟨pasteur-01963025⟩



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