To report our single centre experience in treating 4 children affected by childhood primary central nervous system vasculitis (cPACNS) using mycophenolate mofetil (MMF).

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/m² BID, half-dose for the first 10–15 days followed by full-dose).

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.

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.