title: Giant cell hepatitis with autoimmune hemolytic anemia in children: proposal for therapeutic approach.
abstract: OBJECTIVE
  Giant cell hepatitis (GCH) with autoimmune hemolytic anemia (AIHA) is a rare, progressive disorder in infants and young children. The disease is aggressive and may lead to liver or multiorgan failure with fatal prognosis. Therapy with anti-CD20 monoclonal antibody, rituximab (Rtx), proved effective. The primary objective of the study was to evaluate therapy for severe GCH with AIHA.
  
  
  METHODS
  We report on 5 cases of severe GCH with AIHA treated in our department between 2006 and 2011.
  
  
  RESULTS
  The median age of the children was 7 months (2-12 months), follow-up lasted 44 months (12-78 months), median (min-max), and the main observed symptoms were jaundice and hepatosplenomegaly. All of the children had positive direct Coombs test and biopsy-proven giant cell transformation of hepatocytes. Liver failure was observed in 3 children. First-line therapy (prednisone, azathioprine) proved ineffective in all but 1 of the patients, who initially responded to the treatment but relapsed after 4 months. The child subsequently developed hemophagocytic lymphohistiocytosis and died 2 months after hemopoietic stem cell transplantation. Four remaining patients finally achieved complete remission after 4 to 6 doses of Rtx.
  
  
  CONCLUSIONS
  We propose Rtx as the treatment of choice for severe GCH with AIHA in the early stages of the disease, provided steroids and azathioprine are ineffective.
authors.affiliation: *Department of Gastroenterology, Hepatology, and Eating Disorders, Children's Memorial Health Institute, Warsaw †Department of Hemato-Oncology, Children's Hospital, Kielce ‡Department of Oncology, Children's Memorial Health Institute §Department of Pediatrics, Hematology and Oncology, Medical University of Warsaw, Poland.
authors.forename: Agnieszka
authors.initials: A
authors.lastname: Bakula
authors.forename: Piotr
authors.initials: P
authors.lastname: Socha
authors.forename: Maja
authors.initials: M
authors.lastname: Klaudel-Dreszler
authors.forename: Grazyna
authors.initials: G
authors.lastname: Karolczyk
authors.forename: Malgorzata
authors.initials: M
authors.lastname: Wozniak
authors.forename: Olga
authors.initials: O
authors.lastname: Rutynowska-Pronicka
authors.forename: Michal
authors.initials: M
authors.lastname: Matysiak
mesh_terms: D000744:Anemia, Hemolytic, Autoimmune / Q000150:complications*; D058846:Antibodies, Monoclonal, Murine-Derived / Q000008:administration & dosage / Q000627:therapeutic use*; D001706:Biopsy; D003298:Coombs Test; D005260:Female; D015726:Giant Cells / Q000473:pathology*; D006505:Hepatitis / Q000188:drug therapy* / Q000276:immunology / Q000473:pathology; D022781:Hepatocytes / Q000473:pathology*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D008297:Male; D011379:Prognosis; D012074:Remission Induction; D000069283:Rituximab; D016896:Treatment Outcome
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D007166:Immunosuppressive Agents; D000069283:Rituximab