Objective: To assess the use of mycophenolate mofetil (MMF) in the treatment of refractory primary angiitis of the CNS in childhood (cPACNS).
Methods: A retrospective chart review was performed in patients with cPACNS who were treated with MMF following failure of a combination of corticosteroids and another immunosuppressant.
Results: Three patients from two centres were included in this study. The age of onset of disease was 5, 6 and 9 years. All the patients improved when treated with MMF, such that the dose of corticosteroids could be weaned or stopped.
Conclusions: MMF should be considered for maintenance treatment in the management of patients with cPACNS refractory to the combination of corticosteroids and first-line immunosuppressive agents.