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A child with severe juvenile dermatomyositis treated with ruxolitinib

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Aeschlimann, Florence | Frémond, Marie-Louise | Duffy, Darragh | Rice, Gillian | Charuel, Jean-Luc | Bondet, Vincent | Saire, Elsa | Neven, Bénédicte | Bodemer, Christine | Balu, Laurent | Gitiaux, Cyril | Crow, Yanick | Bader-Meunier, Brigitte

Edité par CCSD ; Oxford University Press -

International audience. 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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