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

Cases J. 2009 Aug 6;2:6609. doi: 10.1186/1757-1626-0002-0000006609.


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.