title: Successful treatment with rituximab and mycophenolate mofetil of refractory autoimmune hemolytic anemia post-hematopoietic stem cell transplant for dyskeratosis congenita due to TINF2 mutation.
abstract: AIHA following allogeneic HSCT is appearing more frequently in the literature. It occurs as a result of donor cell-derived antibodies targeting donor red cell antigens. Little guidance exists on the management of such patients, particularly in the pediatric setting. First-line conventional treatment is corticosteroids and/or immunoglobulin therapy with monoclonal antibody therapy reserved for treatment failure. We report our experience of a child refractory to immunoglobulin and steroid therapy who required several infusions of rituximab and immunomodulatory therapy to obtain a clinically significant response.
authors.affiliation: Department of Haematology, Our Lady's Children's Hospital, Dublin, Ireland.
authors.forename: Niall
authors.initials: N
authors.lastname: O'Connell
authors.forename: Matthew
authors.initials: M
authors.lastname: Goodyer
authors.forename: Mary
authors.initials: M
authors.lastname: Gleeson
authors.forename: Lorna
authors.initials: L
authors.lastname: Storey
authors.forename: Martina
authors.initials: M
authors.lastname: Williams
authors.forename: Melanie
authors.initials: M
authors.lastname: Cotter
authors.forename: Aengus
authors.initials: A
authors.lastname: O'Marcaigh
authors.forename: Owen
authors.initials: O
authors.lastname: Smith
mesh_terms: D000744:Anemia, Hemolytic, Autoimmune / Q000188:drug therapy*; D058846:Antibodies, Monoclonal, Murine-Derived / Q000008:administration & dosage*; D002675:Child, Preschool; D019871:Dyskeratosis Congenita / Q000235:genetics / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007155:Immunologic Factors / Q000008:administration & dosage*; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D008297:Male; D009154:Mutation; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000031:analogs & derivatives*; D000069283:Rituximab; D034501:Telomere-Binding Proteins / Q000235:genetics; D016896:Treatment Outcome
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D007155:Immunologic Factors; D007166:Immunosuppressive Agents; C440868:TINF2 protein, human; D034501:Telomere-Binding Proteins; D000069283:Rituximab; D009173:Mycophenolic Acid
keywords: anemia; hematopoietic stem cell transplant; mycophenolate mofetil; rituximab