Rituximab therapy for multisystem autoimmune diseases in pediatric patients.
OBJECTIVE
To evaluate the effects of 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 (CNS) involvement, were treated with four weekly infusions of rituximab. Their clinical and laboratory responses were evaluated.


RESULTS
Each of the patients had improvement in clinical symptoms and laboratory parameters. One patient with autoimmune cytopenias and autoimmune CNS and peripheral nervous system disease had resolution of the cytopenias and marked improvement in neurologic symptoms; he currently receives no immunosuppressive medications. Two half-siblings with lymphoplasmacytic colitis, pulmonary nodules, and CNS disease had 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 and reduced seizure frequency. There were no serious adverse events.


CONCLUSIONS
The biologic response modifier rituximab, designed to eliminate B lymphocytes, was safe and effective in four pediatric patients with multisystem autoimmune disorders. It appears to be beneficial in autoimmune conditions presumably mediated by a variety of B-cell-related mechanisms, and may decrease or eliminate the need for other immunosuppressive medications.
Binstadt
Bryce A
BA
Rheumatology Program, Division of Immunology, Children's Hospital Boston, Massachusetts 02115, USA.
Caldas
Alvaro M C
AM
Turvey
Stuart E
SE
Stone
Kelley D
KD
Weinstein
Howard J
HJ
Jackson
Jean
J
Fuhlbrigge
Robert C
RC
Sundel
Robert P
RP
D000293:Adolescent; D000906:Antibodies / Q000276:immunology; D000911:Antibodies, Monoclonal / Q000008:administration & dosage / Q000627:therapeutic use*; D058846:Antibodies, Monoclonal, Murine-Derived; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D001327:Autoimmune Diseases / Q000150:complications / Q000276:immunology / Q000628:therapy*; D001991:Bronchitis / Q000150:complications; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007964:Leukocytosis / Q000150:complications* / Q000188:drug therapy*; D008297:Male; D009503:Neutropenia / Q000150:complications / Q000188:drug therapy*; D010523:Peripheral Nervous System Diseases / Q000150:complications* / Q000188:drug therapy*; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000150:complications / Q000188:drug therapy* / Q000276:immunology; D000069283:Rituximab; D012852:Sinusitis / Q000150:complications
D000906:Antibodies; D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D000970:Antineoplastic Agents; D000069283:Rituximab