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Treatment of Hepatitis-Associated Aplastic Anemia with Antithymocyte Globulin and Cyclosporine

Treatment of Hepatitis-Associated Aplastic Anemia with Antithymocyte Globulin and Cyclosporine

Blood 100(11): Abstract No 3514, November 16

H-AA is a variant of acquired aplastic anemia in which AA follows an episode of severe hepatitis. Although patients with H-AA were previously thought to have extremely poor prognosis, recent studies have shown favorable responses to intensive immunosuppressive therapy (IST). The aim of this study was to determine the IST response rate and survival rate of patients with H-AA. Between Jan 1993 and Sep 2001, 291 children with newly diagnosed AA were enrolled into two prospective multicenter trials. All of them received ATG (Lymphoglobulin, 15mg/kg/day, days 1 - 5) and CyA (6mg/kg/day, days 1 - 180). H-AA was defined as AA developed within 6 months after a documented episode of hepatitis. AA was associated with hepatitis in 42 patients (14%; age, 1-18 years, median age, 11 years, male to female ratio, 2 : 1). The virus causing the hepatitis was unkown in all of the patients. All but two patients fulfilled the criteria for severe disease. Thirty-one of 42 patients received G-CSF during follow - up. Six months after the start of ATG therapy, complete response was observed in 12 patients (27%) and partial response was observed in 17 (40%), for an overall response rate of 69%. Additional 2 patients became transfusion-independent between 6 and 12 months after treatment. Of the 31 patients with responses, no one had relapse after an initial response. There were four deaths, the causes of which were cytomegalovirus (CMV) - interstitial pneumonitis (2), bacteremia (1), and fungal pneumonia (1). Six non - responders received bone marrow transplantation (BMT) from an HLA - matched unrelated donor, all of whom are alive and well. New clonal abnormalitiy of del (13) appeared in one patient 18 months after the diagnosis of H-AA, in whom distinctive morphologic features of myelodysplastic syndrome were not found. He is alive and transfusion - independent 85 months after initial diagnosis.Actuarial 8 - year survival was 90% (95%CI; 85% - 95%), which reflects the results of salvage therapy using alternative donor BMT as well as the initial IST. It is noteworthy that CMV - pneumonitis developed only in patients with H-AA and none of 249 patients with other etiologies had this complication after IST.

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Accession: 035980495

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