CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS Rituximab for the treatment of refractory autoimmune hemolytic anemia in children

Autoimmune hemolytic anemia (AIHA) in children is sometimes characterized by a severe course, requiring prolonged ad-ministration of immunosuppressive therapy. Rituximab is able to cause selec-tive in vivo destruction of B lymphocytes, with abrogation of antibody production. In a prospective study, we have evaluated the use of rituximab for the treatment of AIHA resistant to conventional treatment. Fifteen children with AIHA were given rituximab, 375 mg/m2/dose for a median of 3 weekly doses. All patients had previ-ously received 2 or more courses of immu-nosuppressive therapy; 2 patients had undergone splenectomy. After complet-ing treatment, all children received intra-venous immunoglobulin for 6 months. Treatment was well tolerated. With a me-dian follow-up of 13 months, 13 patients (87%) responded, whereas 2 patients did not show any improvement. Median hemo-globin levels increased from 7.7 g/dL to a 2-month posttreatment level of 11.8 g/dL (P <.001). Median absolute reticulocyte counts decreased from 236 to 109 109/L (P <.01). An increase in platelet count was observed in patients with concomi-tant thrombocytopenia (Evans syndrome). Three responder patients had relapse, 7, 8, and 10 months after rituximab infusion, respectively. All 3 children received a second course of rituximab, again achiev-ing disease remission. Our data indicate that rituximab is both safe and effective in reducing or even abolishing hemolysis in children with AIHA and that a sustained response can be achieved in the majority of cases. Disease may recur, but a sec-ond treatment course may be successful in controlling the disease. (Blood. 2003