Outcome of children with aplastic anemia treated with immunosuppressive therapy.

Background: Immunosuppressive therapy (IST) is the alternative treatment in children with aplastic anemia (AA) who do not have an HLA-matched sibling. The aim of this study is to evaluate the outcome of children with AA treated with IST.

Methods: We retrospectively reviewed the hospital records of children with AA from 1984 to 2004, treated at our institution with antithymocyte globulin (ATG), cyclosporine (CS), and short course of prednisone.

Result: Forty-two patients were treated with IST (24 boys, 18 girls); of whom 26% received G-CSF. The median age at diagnosis was 8.5 years. Sixty-nine, 19, and 12% were diagnosed with severe, very severe, and moderate AA, respectively. Twenty-one percent had hepatitis-associated AA. Median follow-up time was 53.3 months. Sixty-two percent had complete response; 19% had partial response. Two patients relapsed and received a second course of ATG; both had a partial response. The actuarial 5 years survival rate was 67.5%. Two patients developed myelodysplastic syndrome (MDS); both received long-term G-CSF and had partial response after two courses of IST. Fifteen percent of survivors had significant hypertension which persisted after CS was discontinued.

Conclusions: This study shows promising response in children with AA treated with IST; however, the outcome was inferior to our institutional results with hematopoietic stem cell transplantation from a sibling donor. Hypertension and MDS are late complications. Longer follow-up, larger cohorts, and prospective studies are warranted to evaluate late complications and risk factors.