%0 Journal Article %T JAK inhibitors are effective in a subset of patients with juvenile dermatomyositis: a monocentric retrospective study %+ Service d'immuno-hématologie pédiatrique [CHU Necker] %+ Human genetics of infectious diseases : Mendelian predisposition (Equipe Inserm U1163) %+ CHU Necker - Enfants Malades [AP-HP] %+ Université Paris Cité (UPCité) %+ Institut Mondor de Recherche Biomédicale (IMRB) %+ Département de pathologie [Mondor] %+ Service de dermatologie [CHU Necker] %+ Laboratory of neurogenetics and neuroinflammation (Equipe Inserm U1163) %+ AP-HP Hôpital universitaire Robert-Debré [Paris] %+ Imagine - Institut des maladies génétiques (IHU) (Imagine - U1163) %+ CHU Trousseau [APHP] %+ Laboratoire de Chimie et de Biochimie Pharmacologiques et Toxicologiques (LCBPT - UMR 8601) %+ Immunologie Translationnelle - Translational Immunology lab %+ CHU Pitié-Salpêtrière [AP-HP] %A Voyer, Tom Le %A Gitiaux, Cyril %A Authier, François-Jérôme %A Bodemer, Christine %A Melki, Isabelle %A Quartier, Pierre %A Aeschlimann, Florence %A Isapof, Arnaud %A Herbeuval, Jean Philippe %A Bondet, Vincent %A Charuel, Jean-Luc %A Frémond, Marie-Louise %A Duffy, Darragh %A Rodero, Mathieu P %A Bader-Meunier, Brigitte %< avec comité de lecture %@ 1462-0324 %J Rheumatology %I Oxford University Press (OUP) %P keab116 %8 2021-02-12 %D 2021 %R 10.1093/rheumatology/keab116 %M 33576769 %K Janus-kinase inhibitor %K juvenile dermatomyositis %K type 1 interferon %Z Life Sciences [q-bio]/ImmunologyJournal articles %X ObjectiveTo evaluate the efficacy and safety of Janus kinase inhibitors (JAKis) in JDM.MethodsWe conducted a single-centre retrospective study of patients with JDM treated by JAKi with a follow-up of at least 6 months. Proportion of clinically inactive disease (CID) within 6 months of JAKi initiation was evaluated using PRINTO criteria and skin Disease Activity Score. Serum IFN-α concentration was measured by Simoa assay.ResultsNine refractory and one new-onset patients with JDM treated with ruxolitinib (n = 7) or baricitinib (n = 3) were included. The main indications for treatment were refractory muscle involvement (n = 8) and ulcerative skin disease (n = 2). CID was achieved in 5/10 patients (two/two anti-MDA5, three/four anti-NXP2, zero/three anti-TIF1γ-positive patients) within 6 months of JAKi introduction. All responders could withdraw plasmatic exchange, immunoadsorption and other immunosuppressive drugs. The mean daily steroid dose decreased from 1.1 mg/kg (range 0.35–2 mg/kg/d) to 0.1 (range, 0–0.3, P = 0.008) in patients achieving CID, and was stopped in two. Serum IFN-α concentrations were elevated in all patients at the time of treatment initiation and normalized in both responder and non-responder. A muscle biopsy repeated in one patient 26 months after the initiation of JAKi, showed a complete restoration of muscle endomysial microvascular bed. Herpes zoster and skin abscesses developed in three and two patients, respectively.ConclusionJAKis resulted in a CID in a subset of new-onset or refractory patients with JDM and may dramatically reverse severe muscle vasculopathy. Overall tolerance was good except for a high rate of herpes zoster infection. %G English %2 https://pasteur.hal.science/pasteur-03236464/document %2 https://pasteur.hal.science/pasteur-03236464/file/JAKiDMRheumatologyREVISEDFINAL.pdf %L pasteur-03236464 %U https://pasteur.hal.science/pasteur-03236464 %~ PASTEUR %~ CNRS %~ APHP %~ IMRB %~ UPEC %~ INC-CNRS %~ CHU-UNIV-PARIS5 %~ SORBONNE-UNIVERSITE %~ SORBONNE-UNIV %~ SU-MEDECINE %~ SU-MED %~ UNIV-PARIS %~ UNIVERSITE-PARIS %~ UP-SANTE %~ UP-SCIENCES %~ TEST-HALCNRS %~ SU-TI %~ ALLIANCE-SU