title: 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 x 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.
authors.affiliation: Oncoematologia Pediatrica, IRCCS Policlinico San Matteo, Pavia, Italy.
authors.forename: Marco
authors.initials: M
authors.lastname: Zecca
authors.forename: Bruno
authors.initials: B
authors.lastname: Nobili
authors.forename: Ugo
authors.initials: U
authors.lastname: Ramenghi
authors.forename: Silverio
authors.initials: S
authors.lastname: Perrotta
authors.forename: Giovanni
authors.initials: G
authors.lastname: Amendola
authors.forename: Pasquale
authors.initials: P
authors.lastname: Rosito
authors.forename: Momcilo
authors.initials: M
authors.lastname: Jankovic
authors.forename: Paolo
authors.initials: P
authors.lastname: Pierani
authors.forename: Piero
authors.initials: P
authors.lastname: De Stefano
authors.forename: Mario Regazzi
authors.initials: MR
authors.lastname: Bonora
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
mesh_terms: D000293:Adolescent; D000743:Anemia, Hemolytic / Q000097:blood / Q000188:drug therapy* / Q000276:immunology; D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D058846:Antibodies, Monoclonal, Murine-Derived; D001327:Autoimmune Diseases / Q000188:drug therapy*; D001402:B-Lymphocytes / Q000187:drug effects / Q000276:immunology*; D002648:Child; D005260:Female; D006454:Hemoglobins / Q000378:metabolism; D006801:Humans; D007223:Infant; D008297:Male; D017701:Reticulocyte Count; D000069283:Rituximab; D016896:Treatment Outcome
chemical_list: D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D006454:Hemoglobins; D000069283:Rituximab