OBJECTIVETo study the effectiveness and safety of immunosuppressive therapy (IST) in the treatment of childhood aplastic anemia (AA) and to study the main factors influencing the effectiveness.

METHODSThe clinical data of 55 children with severe aplastic anemia (SAA) and 51 children with chronic aplastic anemia (CAA) were retrospectively analyzed. All patients received IST from January 2007 to December 2010.

RESULTSIn children with CAA, the effective rate of antithymocyte globulin (ATG) plus cyclosporine A(CsA) combination therapy was significantly higher than that of CsA alone (80% vs 44%; P<0.05); in children with SAA, the effective rate of ATG plus CsA combination therapy was also significantly higher than that of CsA alone (75% vs 40%; P<0.05). No patients developed clonal disease such as myelodysplastic syndrome, paroxysmal nocturn hemoglobinuria or acute myelocytic leukemia. In patients treated with the ATG plus CsA combination therapy, the response rate was relatively high for children whose disease course was less than six months, bone marrow hematopoietic area was more than 40%, had no severe infections, and experienced granulocyte colony stimulating factor (G-CSF) reaction during the early treatment; however, it was not related to AA subtypes and age.

CONCLUSIONSATG plus CsA combination therapy is effective and safe in the treatment of childhood AA. The disease course, bone marrow hematopoietic area, severe infections and G-CSF reaction to early treatment are the main factors influencing the therapeutic effects.