Pediatric Rheumatology Online Journal → August 2003 → Newer Treatments → Anti-CD20 Monoclonal Antibody (Rituximab) → Abstract #162

RITUXIMAB THERAPY FOR MULTISYSTEM AUTOIMMUNE DISEASES IN PEDIATRIC PATIENTS

B. A. Binstadt,¹ H. J. Weinstein,² J. Jackson,^1,3 R. C. Fuhlbrigge,¹ R. P. Sundel.¹

¹Rheumatology Program, Children's Hospital Boston, Boston, MA; ²Pediatric Hematology-Oncology Unit, MassGeneral Hospital for Children, Boston, MA; ³Department of Rheumatology, Brigham and Women's Hospital, Boston, MA

Objective: To evaluate the effects of B lymphocyte depletion with rituximab, anti-CD20 monoclonal antibody, on the disease course in pediatric patients with multisystem autoimmune diseases.
Methods: Four patients with multisystem autoimmune diseases refractory to conventional immunosuppressive medications, each with central nervous system involvement, were treated with four weekly infusions of rituximab. Their clinical and laboratory responses were evaluated.
Results: Each of the patients experienced improvement in clinical symptoms and laboratory signs. One patient with autoimmune cytopenias and autoimmune central and peripheral nervous system disease experienced resolution of the cytopenias and marked improvement in neurologic symptoms; he is currently on no immunosuppressive medications. Two half-siblings with plasmacytoid colitis, pulmonary nodules, and central nervous system disease experienced improvement of their symptoms. A fourth patient with chorea and seizures secondary to primary antiphospholipid antibody syndrome had improvement in fine and gross motor function, reduced seizure frequency, and is weaning off anticonvulsant medications. There were no serious adverse events.
Conclusions: The biologic response modifier rituximab, designed to eliminate B lymphocytes, may be safe and effective in pediatric patients with multisystem autoimmune disorders. It appears to be beneficial in autoimmune conditions mediated by a variety of T- and B-cell related mechanisms, and may decrease or eliminate the need for other immunosuppressive medications.