title: Biologic therapy in refractory chronic non-bacterial osteomyelitis of childhood.
abstract: OBJECTIVE
  To date there is no uniformly effective treatment for either chronic recurrent multifocal osteomyelitis (CRMO) or synovitis, acne, pustulosis, hyperostosis and osteitis (SAPHO) syndrome. We report on our clinical experience of using biologic therapy to treat children with these conditions.
  
  
  METHODS
  Retrospective descriptive case series of four children with refractory disease treated with biologics. Disease activity was assessed at predetermined time points (T = 0, T = 6 weeks and T = 12 months after the start of biologic therapy, and at latest follow-up) using a combination of clinical examination and radiological findings: a 10 cm pain and physician visual analogue scale; the Childhood Health Assessment Questionnaire as an assessment of disability; and changes in markers of systemic inflammation.
  
  
  RESULTS
  There was an initial improvement in all parameters assessed for all three children treated with TNF-alpha blockade, although the third case had to discontinue the therapy due to a suspected (but unconfirmed) fungal skin infection. Anakinra treatment alleviated the symptoms in the fourth patient at 6 weeks, but there was no sustained response to treatment at 1-year follow-up.
  
  
  CONCLUSIONS
  We present our preliminary experience of using biological therapies to treat children with CRMO and SAPHO in conjunction with other immunosuppression. Further studies are needed to establish the role of these therapies in refractory CRMO and SAPHO.
authors.affiliation: Department of Rheumatology, Institute of Child Health and Great Ormond Street Hospital, London WC1N 1EH, UK. d.eleftheriou@ich.ucl.ac.uk
authors.forename: Despina
authors.initials: D
authors.lastname: Eleftheriou
authors.forename: Tommy
authors.initials: T
authors.lastname: Gerschman
authors.forename: Neil
authors.initials: N
authors.lastname: Sebire
authors.forename: Patricia
authors.initials: P
authors.lastname: Woo
authors.forename: Clarissa A
authors.initials: CA
authors.lastname: Pilkington
authors.forename: Paul A
authors.initials: PA
authors.lastname: Brogan
mesh_terms: D020083:Acquired Hyperostosis Syndrome / Q000188:drug therapy* / Q000503:physiopathology; D018501:Antirheumatic Agents / Q000627:therapeutic use*; D001691:Biological Therapy / Q000379:methods; D002648:Child; D002908:Chronic Disease; D006801:Humans; D007223:Infant; D053590:Interleukin 1 Receptor Antagonist Protein / Q000627:therapeutic use*; D010019:Osteomyelitis / Q000188:drug therapy* / Q000503:physiopathology; D012189:Retrospective Studies; D016896:Treatment Outcome; D014409:Tumor Necrosis Factor-alpha / Q000037:antagonists & inhibitors* / Q000627:therapeutic use
chemical_list: D018501:Antirheumatic Agents; D053590:Interleukin 1 Receptor Antagonist Protein; D014409:Tumor Necrosis Factor-alpha