Rituximab for the treatment of refractory autoimmune hemolytic anemia in children

Abstract

Autoimmune hemolytic anemia (AIHA) in children is sometimes characterized by a severe course, requiring prolonged administration of immunosuppressive therapy. Rituximab is able to cause selective 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/m 2 /dose for a median of 3 weekly doses. All patients had previously received 2 or more courses of immunosuppressive therapy; 2 patients had undergone splenectomy. After completing treatment, all children received intravenous immunoglobulin for 6 months. Treatment was well tolerated. With a median follow-up of 13 months, 13 patients (87%) responded, whereas 2 patients did not show any improvement. Median hemoglobin 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 ؋ 10 9 /L (P < .01). An increase in platelet count was observed in patients with concomitant 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 achieving 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 second treatment course may be successful in controlling the disease. (Blood. 2003; 101:3857-3861)

Lower dose rituximab is active in adults patients with idiopathic thrombocytopenic purpura

“…Rituximab is an emerging new agent for the treatment of several autoimmune disorders [1][2][3][4][5][6][7][8][9] and, in particular, many reports highlighted the effect of rituximab in idiopathic thrombocytopenic purpura (ITP). [10][11][12][13][14][15][16][17][18] To date, rituximab has been administered with the same schedule proposed for Bcell lymphomas, i.e.…”

Successful treatment of autoimmune hemolytic anemia with rituximab in a child with severe combined immunodeficiency following nonidentical T-cell-depleted bone marrow transplantation

“…[3][4][5] A good response to rituximab has been described in three children with AIHA following HSCT performed for the treatment of mucopolysaccharidosis. [3][4][5] So far, a better response to rituximab could be achieved in patients with cold-agglutinin disease, as compared with warm-autoantibody AIHA. 3,4 At present, this 5-year-old boy is in good clinical condition and free of infectious events.…”

“…6 Rituximab results in prolonged and severe B-cell depletion. [3][4][5] This antibody is increasingly being used for the treatment of AIHA refractory to conventional therapy. [3][4][5] A good response to rituximab has been described in three children with AIHA following HSCT performed for the treatment of mucopolysaccharidosis.…”

“…2 The outcome of severe childhood AIHA following HSCT with ineffective response to conventional immunosuppressive therapy is usually dismal. [2][3][4][5] T-cell precursors may undergo aberrant thymic education after HSCT, contributing to both the immunodeficiency and autoimmunity that characterise chronic GVHD and autoimmune diseases post-HSCT. 6 Rituximab results in prolonged and severe B-cell depletion.…”

“…[3][4][5] This antibody is increasingly being used for the treatment of AIHA refractory to conventional therapy. [3][4][5] A good response to rituximab has been described in three children with AIHA following HSCT performed for the treatment of mucopolysaccharidosis. [3][4][5] So far, a better response to rituximab could be achieved in patients with cold-agglutinin disease, as compared with warm-autoantibody AIHA.…”