Pediatric Rheumatology Online Journal → July 2003 → Childhood Lupus→ Treatment → Abstract #102
                                                                August 2003 → Newer Treatments → Anti-CD20 Monoclonal Antibody (Rituximab) → Abstract #102

USE OF RITUXIMAB IN PEDIATRIC PATIENTS WITH AUTOANTIBODY-MEDIATED BLEEDING DISORDERS ASSOCIATED WITH LUPUS-LIKE OVERLAP SYNDROMES

P. R. Blier,¹ D. Rothman,² P. G. Sprinz.³

¹Pediatrics & Pediatric Rheumatology, Riverbend Medical Group, Chicopee, MA; ²Pediatric Rheumatology, Shriners Hospital for Children, Springfield, MA; ³Pediatric Hematology-Oncology, Baystate Medical Center, Springfield, MA

Certain pathophysiological manifestations of autoimmune disease are mediated by autoantibodies. Depletion of B cells and reduction in the production of pathological antibodies may be helpful in these disorders. Rituximab, a monoclonal antibody against the B cell antigen CD20 developed to treat B cell lymphoma, has been used to treat adult patients with autoimmune hemolytic anemia and thrombocytopenia. We describe two pediatric patients with life-threatening refractory bleeding as part of a lupus-like overlap syndrome who have been treated with rituximab after failing treatment with other immunosuppressive regimens, including long term high dose corticosteroids.
The first patient is a 13 year old girl with an SLE/scleroderma overlap who has recurrent profound thrombocytopenia. Methotrexate, IV gammaglobulin, and methylprednisolone were ineffective or gave only transient improvement. After four weekly infusions of rituximab at 375 mg/m2, her platelet count gradually returned to normal and remained stable for 6 months with no bleeding episodes. She was able to taper or eliminate other medications. No complications attributable to rituximab infusion developed. After 6 months, platelet count again fell to 1x10(6)/L. She has been started on a second cycle of rituximab.
The second patient is an 8 year old girl with an SLE-like syndrome and positive lupus anticoagulant, whose severe mucous membrane bleeding appears to be associated with acquired inhibitors of factor VIII/vWF. Azathioprine and corticosteroids have provided only partial relief. She received four infusions of rituximab along with pulse IV methylprednisolone; infusions have been well tolerated and coagulation parameters have normalized. There have been no infectious complications up to this time.
We conclude that rituximab may be a useful therapeutic option in children with autoimmune bleeding disorders who have failed conventional therapy. Further study is needed to better define its use in this population.