title: Rituximab for the treatment of post-bone marrow transplantation refractory hemolytic anemia in a child with Omenn's syndrome.
abstract: Omenn's syndrome is a rare severe combined immunodeficiency that kills affected subjects before the end of the first year of life unless patients are treated with bone marrow transplantation (BMT). Unfortunately, post-BMT patients may develop autoimmune diseases, such as autoimmune hemolytic anemia (AIHA), which sometimes fails to respond to standard therapies. Rituximab is a chimeric, human, immunoglobulin G1/k monoclonal antibody specific for the CD20 antigen expressed on the surface of B lymphocytes. Rituximab is currently only labeled for treatment of B-cell lymphoproliferative disorders, such as B-cell non-Hodgkin's lymphoma and follicular lymphoma; however, it is also employed in the treatment of a variety of disorders mediated by auto-antibodies, such as AIHA and transplant-related autoimmune disorders. Herein, we describe the case of a 23-month-old male child with Omenn's syndrome, who had undergone BMT and was successfully treated with rituximab (375 mg/m(2) intravenously, weekly for three times) for refractory post-BMT hemolytic anemia. Our findings evidence that rituximab should be considered for treatment of post-BMT AIHA refractory to traditional therapy also in children with primary immunodeficiencies; furthermore, rituximab might represent a means to obtain remissions without the toxic effects associated with corticosteroid and immunosuppressive agents.
authors.affiliation: Paediatric Therapy, Operative Unit of Genetic and Immunology Paediatrics, University of Messina, Contrada Annunziata, 98168 Messina, Italy.
authors.forename: Briuglia
authors.initials: B
authors.lastname: Silvana
authors.forename: La Mazza
authors.initials: LM
authors.lastname: Antonella
authors.forename: Piraino
authors.initials: P
authors.lastname: Basilia
authors.forename: Domenico
authors.initials: D
authors.lastname: Trombetta
authors.forename: Antonella
authors.initials: A
authors.lastname: Saija
authors.forename: Carmelo
authors.initials: C
authors.lastname: Salpietro
mesh_terms: D000743:Anemia, Hemolytic / Q000188:drug therapy*; D000753:Anemia, Refractory / Q000188:drug therapy*; D000911:Antibodies, Monoclonal / Q000008:administration & dosage / Q000627:therapeutic use*; D058846:Antibodies, Monoclonal, Murine-Derived; D018951:Antigens, CD20; D016026:Bone Marrow Transplantation / Q000009:adverse effects*; D005500:Follow-Up Studies; D006801:Humans; D007155:Immunologic Factors / Q000008:administration & dosage / Q000627:therapeutic use*; D007223:Infant; D007275:Injections, Intravenous; D008297:Male; D011183:Postoperative Complications; D000069283:Rituximab; D016511:Severe Combined Immunodeficiency / Q000601:surgery*; D013577:Syndrome
chemical_list: D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D018951:Antigens, CD20; D007155:Immunologic Factors; D000069283:Rituximab