Mycophenolate Mofetil as First-line Induction and Maintenance Therapy with cPACNS: A Case Report

Aim and background: To report our experience in successfully treating a child with primary central nervous system vasculitis (cPACNS) using mycophenolate mofetil (MMF) as a first-line agent for both induction and maintenance therapy.This is probably the first case report from India in which MMF has been used as a first-line agent in a child with cPACNS.Case description: A previously healthy 7-year-old boy presented with nausea, vomiting, and left-sided weakness.Magnetic resonance imaging revealed multiple infarcts and cerebellar mass effect, with angiographic findings suggestive of vasculitis.Extensive workup excluded secondary causes.He was diagnosed with cPACNS and treated with pulse steroids, followed by oral steroids and MMF for induction and maintenance therapy.The patient showed continuous gradual neurological improvement, and a 3-month follow-up and subsequently 2-year follow-up showed no new infarcts.Mycophenolate mofetil was well tolerated.Conclusion: This case underscores the utility of oral MMF as a first-line agent for both induction and maintenance therapy in cPACNS, especially in settings where regular hospital visits are challenging for intravenous cyclophosphamide.Clinical significance: Early diagnosis and a tailored treatment approach using MMF can provide effective disease control and minimize hospital burden in pediatric cPACNS, improving outcomes in real-world scenarios.