title: Mycophenolate mofetil as induction and long-term maintaining treatment in childhood: Primary angiitis of the central nervous system.
abstract: OBJECTIVE
  To report our single centre experience in treating 4 children affected by childhood primary central nervous system vasculitis (cPACNS) using mycophenolate mofetil (MMF).
  
  
  METHODS
  From December 2011 to August 2015, 4 patients (3 males; age range: 9 months-13 years) affected by cPACNS were collected. Enrolled children received the following treatment protocol: acetylsalicylic acid and/or anticoagulant therapy with low molecular weight heparin (LMWH) 100 U/k BID replaced by acenocoumarol; methyl-prednisolone (30mg/kg/day for 3-5 days) followed by prednisone (2mg/kg/day), tapered and discontinued over 7-8 months; MMF used for induction therapy and subsequent maintenance phase (750-1000mg/m2BID, half-dose for the first 10-15 days followed by full-dose).
  
  
  RESULTS
  In all children, no relapse of cerebral vasculitis occurred during the whole follow-up period and all of them improved while in MMF treatment. Magnetic resonance imaging (MRI) and magnetic resonance angiography (MRA), performed at 6, 9 or 12 months intervals, showed no progression or even improvement of the typical radiological findings. Medium period of MMF treatment was 29 months (range: 10-42 months). No major drug-related adverse events were documented.
  
  
  CONCLUSIONS
  We report for the first time on the efficacy and safety of MMF in the induction and maintenance of clinical remission in cPACNS. Our single centre experience of MMF use in treating cPACNS seems represent an appealing, alternative and safe option in this clinical setting over a long-term follow-up.
authors.affiliation: Paediatric Neurology Unit, NEUROFARBA Department, Anna Meyer Children's Hospital, University of Florence, 50139 Firenze, Italy.
authors.forename: Anna
authors.initials: A
authors.lastname: Rosati
authors.affiliation: Paediatric Neurology Unit, NEUROFARBA Department, Anna Meyer Children's Hospital, University of Florence, 50139 Firenze, Italy.
authors.forename: Alessandra
authors.initials: A
authors.lastname: Cosi
authors.affiliation: Radiology Unit, Anna Meyer Children's Hospital, 50139 Firenze, Italy.
authors.forename: Massimo
authors.initials: M
authors.lastname: Basile
authors.affiliation: Rheumatology Unit-Paediatric Section, NEUROFARBA Department, Anna Meyer Children's Hospital, University of Florence, Pieraccini street, # 24, 50139 Firenze, Italy.
authors.forename: Alice
authors.initials: A
authors.lastname: Brambilla
authors.affiliation: Paediatric Neurology Unit, NEUROFARBA Department, Anna Meyer Children's Hospital, University of Florence, 50139 Firenze, Italy.
authors.forename: Renzo
authors.initials: R
authors.lastname: Guerrini
authors.affiliation: Rheumatology Unit-Paediatric Section, NEUROFARBA Department, Anna Meyer Children's Hospital, University of Florence, Pieraccini street, # 24, 50139 Firenze, Italy.
authors.forename: Rolando
authors.initials: R
authors.lastname: Cimaz
authors.affiliation: Rheumatology Unit-Paediatric Section, NEUROFARBA Department, Anna Meyer Children's Hospital, University of Florence, Pieraccini street, # 24, 50139 Firenze, Italy. Electronic address: gabriele.simonini@unifi.it.
authors.forename: Gabriele
authors.initials: G
authors.lastname: Simonini
mesh_terms: D000293:Adolescent; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D009173:Mycophenolic Acid / Q000627:therapeutic use*; D020521:Stroke / Q000000981:diagnostic imaging / Q000209:etiology; D020293:Vasculitis, Central Nervous System / Q000150:complications / Q000000981:diagnostic imaging / Q000188:drug therapy*
chemical_list: D000893:Anti-Inflammatory Agents; D009173:Mycophenolic Acid
keywords: Central nervous system; Childhood; Mycophenolate mofetil; Primary angiitis