title: Anti-CD20 treatment of giant cell hepatitis with autoimmune hemolytic anemia.
abstract: Giant cell hepatitis with autoimmune hemolytic anemia (GCH-AHA) is a rare autoimmune disease of infancy characterized by severe liver disease associated with Coombs-positive hemolytic anemia. We recently showed that GCH-AHA is probably caused by a humoral immune mechanism. Such data support the use of rituximab, an anti-CD-20 monoclonal antibody specifically targeting B lymphocytes, as a treatment for GCH-AHA. We describe here the detailed clinical evolution of 4 children with GCH-AHA who showed a complete response to rituximab. All patients shared a severe course of the disease with poor control on standard and aggressive immunosuppression. Rituximab was well tolerated, and no side effects or infections were registered. Several doses were needed to induce remission, and 5 to 11 additional maintenance injections were necessary in the 2 more severe cases. Weaning from corticosteroids was achieved in all subjects. A steroid-sparing effect was noted in the 3 children who started rituximab early in the course of the disease. Overall, we show here that there is a strong rationale for treating GCH-AHA with rituximab. Early treatment could reduce the use of corticosteroids. Nevertheless, short-term steroids should be initially associated with rituximab to account for autoantibodies' half-life. Repeated injections are needed to treat and prevent relapses, but the best frequency and duration of treatment remain to be defined.
authors.affiliation: Departments of Gastroenterology, Hepatology and Nutrition, and m.paganelli@umontreal.ca.
authors.forename: Massimiliano
authors.initials: M
authors.lastname: Paganelli
authors.affiliation: Pathology, Centre Hospitalier Universitaire Sainte-Justine, Université de Montréal, Montreal (QC), Canada;
authors.forename: Natacha
authors.initials: N
authors.lastname: Patey
authors.affiliation: Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois; and Department of Pediatrics, Feinberg Medical School of Northwestern University, Chicago, Illinois.
authors.forename: Lee M
authors.initials: LM
authors.lastname: Bass
authors.affiliation: Departments of Gastroenterology, Hepatology and Nutrition, and.
authors.forename: Fernando
authors.initials: F
authors.lastname: Alvarez
mesh_terms: D000293:Adolescent; D000744:Anemia, Hemolytic, Autoimmune / Q000150:complications / Q000188:drug therapy* / Q000276:immunology*; D058846:Antibodies, Monoclonal, Murine-Derived / Q000494:pharmacology / Q000627:therapeutic use*; D018951:Antigens, CD20 / Q000276:immunology*; D001323:Autoantibodies / Q000276:immunology; D002648:Child; D015726:Giant Cells / Q000276:immunology / Q000473:pathology; D006505:Hepatitis / Q000150:complications / Q000188:drug therapy* / Q000276:immunology*; D006801:Humans; D000069283:Rituximab; D016896:Treatment Outcome
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D018951:Antigens, CD20; D001323:Autoantibodies; D000069283:Rituximab
keywords: autoimmune hemolytic anemia; children; giant cell hepatitis; rituximab