title: Safety and efficacy of rituximab in severe juvenile dermatomyositis: results from 9 patients from the French Autoimmunity and Rituximab registry.
abstract: OBJECTIVE
  To evaluate the safety and efficacy of rituximab (RTX) in juvenile dermatomyositis (JDM) in off-trial patients.
  
  
  METHODS
  We conducted a multicenter prospective study of patients with JDM included in the French Autoimmunity and Rituximab (AIR) registry.
  
  
  RESULTS
  Nine patients with severe JDM were studied. The main indication for RTX treatment was severe and/or refractory muscle involvement (7 patients), severe calcinosis (1 patient), or severe chronic abdominal pain associated with abdominal lipomatosis (1 patient). RTX was associated with corticosteroids, immunosuppressive drugs, and plasma exchange therapy in 9/9, 5/9, and 2/9 patients, respectively. Mild infections of the calcinosis sites occurred in 2 patients and an infusion-related event in 1. Complete clinical response was achieved in 3/6 patients treated with RTX for muscle involvement. In these responders steroid therapy was stopped or tapered to < 15% of the baseline dosage, with no relapse, with a followup ranging from 1.3 to 3 years. Calcinosis did not improve in the 6 affected patients.
  
  
  CONCLUSIONS
  This small series suggests that rituximab may be effective for treating muscle and skin involvement in a small subset of children with severe JDM, and that its safety profile was satisfactory. Further studies are needed to identify predictive factors of response to RTX in patients with severe JDM.
authors.affiliation: Department of Pediatric Immunology and Rheumatology, Paris Descartes University, AP-HP, Necker Hospital, Paris, France. brigitte.bader-meunier@nck.aphp.fr
authors.forename: Brigitte
authors.initials: B
authors.lastname: Bader-Meunier
authors.forename: Hélène
authors.initials: H
authors.lastname: Decaluwe
authors.forename: Christine
authors.initials: C
authors.lastname: Barnerias
authors.forename: Romain
authors.initials: R
authors.lastname: Gherardi
authors.forename: Pierre
authors.initials: P
authors.lastname: Quartier
authors.forename: Albert
authors.initials: A
authors.lastname: Faye
authors.forename: Vincent
authors.initials: V
authors.lastname: Guigonis
authors.forename: Anne
authors.initials: A
authors.lastname: Pagnier
authors.forename: Karine
authors.initials: K
authors.lastname: Brochard
authors.forename: Jean
authors.initials: J
authors.lastname: Sibilia
authors.forename: Jacques-Eric
authors.initials: JE
authors.lastname: Gottenberg
authors.forename: Christine
authors.initials: C
authors.lastname: Bodemer
mesh_terms: D000293:Adolescent; D058846:Antibodies, Monoclonal, Murine-Derived / Q000009:adverse effects* / Q000627:therapeutic use*; D018501:Antirheumatic Agents / Q000009:adverse effects / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003882:Dermatomyositis / Q000188:drug therapy* / Q000453:epidemiology; D005260:Female; D005602:France / Q000453:epidemiology; D006801:Humans; D007223:Infant; D008297:Male; D011446:Prospective Studies; D012042:Registries; D000069283:Rituximab; D012720:Severity of Illness Index; D016896:Treatment Outcome
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D018501:Antirheumatic Agents; D000069283:Rituximab