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

To report on efficacy, tolerability and safety of rituximab in children with refractory autoimmune diseases. 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. Efficacy of rituximab was variable ranging from complete remission to inefficacy. Treatment was safe.