Immunosuppressive therapy using antithymocyte globulin, cyclosporine, and danazol with or without human granulocyte colony-stimulating factor in children with acquired aplastic anemia

A prospective multicenter trial of 119 children 1 to 18 years of age with n ewly diagnosed aplastic anemia (AA) was conducted, comparing treatment usin g antithymocyte globulin (ATG), cyclosporine (CyA), and danazol (DAN) with or without rh(jr-CSF (400 mu g/m(2), day on days 1-90), All children with v ery severe AA received rhG-CSF (VSAA group, n = 50), The other children wer e randomized to receive ATG, CyA, DAN, and rhG-CSF (G-CSF+ group, n = 35) o r ATG, CyA, and DAN without rhG-CSF (G-CSF-group, n = 34), After 6 months, the hematologic response rate was 71%, 55%, and 77% in the VSAA group, G-CS F+ group, and G-CSF- group, respectively. There was no difference in the in cidence of febrile episodes and documented infections between the G-CSF+ an d G-CSF- groups. Bone marrow transplantation (BMT) was attempted in 22 pati ents in whom initial immunosuppressive therapy (IST; n = 18) failed or in w hom a relapse occurred after an initial response (n = 4). Nineteen of the 2 2 patients are alive and well after a median follow-up of 18 months (range, 3 to 66 months) since BMT. The probability of survival at 4 years was 83% +/- 7% in the VSAA group, 91% +/- 5% in the G-CSF+ group, and 93% +/- 6% in the G-CSF- group. Myelodysplastic syndrome (MDS)/acute myeloid leukemia (A ML) developed in one patient in each of the three groups; the overall risk for MDS/AML was 3% +/- 2% at 4 years, Because the results of IST were encou raging, it is suggested that children with AA receive IST as first-line the rapy if there is no human leukocyte antigen-matched sibling donor, (Blood. 2000;96:2049-2054) (C) 2000 by The American Society of Hematology.