A child with severe juvenile dermatomyositis treated with ruxolitinib

Florence Aeschlimann (1) , Marie-Louise Frémond (2, 1) , Darragh Duffy (3) , Gillian Rice (4) , Jean-Luc Charuel (5) , Vincent Bondet (3) , Elsa Saire (1) , Bénédicte Neven (1, 2) , Christine Bodemer (1, 2) , Laurent Balu (6) , Cyril Gitiaux (7, 8) , Yanick Crow (2, 9) , Brigitte Bader-Meunier (1, 2)

1 Service d'immuno-hématologie pédiatrique [CHU Necker]
2 IMAGINE - U1163 - Imagine - Institut des maladies génétiques
3 Immunobiologie des Cellules dendritiques
4 University of Manchester [Manchester]
5 Service d'Immunologie [CHU Pitié-Salpétrière]
6 CHU La Réunion - Centre Hospitalier Universitaire de La Réunion
7 IMRB - Institut Mondor de Recherche Biomédicale
8 CHU Necker - Enfants Malades [AP-HP]
9 University of Edinburgh

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 [...]

Domains: Life Sciences [q-bio]; Immunology

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⟩