Background and 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.
*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.
Address correspondence and reprint requests to Agnieszka Bakula, Department of Gastroenterology, Hepatology, and Eating Disorders, Children's Memorial Health Institute, Al. Dzieci Polskich 20, 04-730 Warsaw, Poland (e-mail: firstname.lastname@example.org).
Received 16 April, 2013
Accepted 25 November, 2013
The authors report no conflicts of interest.