Rituximab for the treatment of refractory pediatric autoimmune diseases: a case series

INTRODUCTION: To report on efficacy, tolerability and safety of rituximab in children with refractory autoimmune diseases. CASE PRESENTATION: Five patients (juvenile dermatomyositis, Wegener's granulomatosis, systemic lupus erythematosus, myasthenia gravis and multiple sclerosis with systemic lupus erythematosus) were treated with rituximab and followed for a median time of 2.5 years. Two patients achieved remission (systemic lupus erythematosus, Wegener's granulomatosis). Three patients had a refractory disease course and underwent autologous stem cell transplantation. Of those, two achieved remission (juvenile dermatomyositis, myasthenia gravis), one died of complications after transplantation (multiple sclerosis/systemic lupus erythematosus). No severe adverse events occurred. CONCLUSION: Efficacy of rituximab was variable ranging from complete remission to inefficacy. Treatment was safe.