Outcome of children with aplastic anemia treated with immunosuppressive therapy

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.

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.

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.

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.