Anti-CD20 monoclonal antibody for the treatment of severe, immune-mediated, pure red cell aplasia and hemolytic anemia.

Immune-mediated, acquired pure red cell aplasia (PRCA) is a rare disorder frequently associated with other autoimmune phenomena. Conventional immunosuppressive treatment is often unsatisfactory. Rituximab is a monoclonal antibody against the CD20 antigen, highly effective for in vivo B-cell depletion. An 18-month-old girl with both severe PRCA and autoimmune hemolytic anemia, refractory to immunosuppressive treatment, received 2 doses of rituximab, 375 mg/m(2) per week. The drug was well tolerated. After anti-CD20 therapy, substitutive treatment with intravenous immunoglobulin was started. The treatment resulted in marked depletion of B cells; a striking rise in reticulocyte count ensued, with increasing hemoglobin levels, finally leading to transfusion independence. The child is now 5 months off-therapy, with normal hemoglobin and reticulocyte levels. This case suggests a role of anti-CD20 monoclonal antibody for treatment of patients with antibody-mediated hematologic disorders. (Blood. 2001;97:3995-3997)