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JAK inhibitors are effective in a subset of patients with juvenile dermatomyositis: a monocentric retrospective study

2021; Oxford University Press; Volume: 60; Issue: 12 Linguagem: Inglês

10.1093/rheumatology/keab116

1462-0332

Tom Le Voyer, Cyril Gitiaux, François‐Jérôme Authier, Christine Bodemer, Isabelle Melki, Pierre Quartier, Florence A. Aeschlimann, Arnaud Isapof, Jean‐Philippe Herbeuval, Vincent Bondet, Jean‐Luc Charuel, Marie‐Louise Frémond, Darragh Duffy, Mathieu P. Rodero, Brigitte Bader‐Meunier,

Immunodeficiency and Autoimmune Disorders

To evaluate the efficacy and safety of Janus kinase inhibitors (JAKis) in JDM.We 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.Nine 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.JAKis 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.