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

Abstract

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.

Figure 1

B-cell depletion in patients 1 (JDM), 2 (WG), 4 (SLE/MG) and 5 (MS/SLE). No clear correlation between B-cell recurrence and disease flares can be seen. In patient 4 (SLE/MG) B-cells were reduced below normal levels due to treatment with cyclophosphamide. JDM, juvenile dermatomyositis; WG, Wegener's granulomatosis; SLE, systemic lupus erythematodes; MG, myasthenia gravis; MS, multiple sclerosis; ASCT, autologous stem cell transplantation; RTX, rituximab.