Definitions
OS was measured from the first day of IST until death or the date last
known alive. Relapse was defined as reduced blood counts that warranted
the reintroduction of full-dose CSA. Clonal evolution was defined as
progression to myelodysplastic syndrome and acute myeloid leukemia. A
PNH clone was considered present if glycophosphatidylinositol hemocytes
exceeded 5% according to flow cytometry data. EFS was measured from the
start of IST until an event (death, relapse, clone evolution or start of
a second therapy for aplastic anemia, either hematopoietic stem cell
transplantation or a second course of IST). CR was defined with a bone
marrow sample showing <5% myeloblasts with normal maturation
of all cell lines and no dysplasia, peripheral blood neutrophil count
≥1×109/L, hemoglobin ≥10 g/dL, and platelet count
≥100×109/L. Partial response (PR) was reflected by
transfusion independence and G-CSF with ANC
≥0.5×109/L, hemoglobin ≥8g/dL, and platelet count
≥20×109/L. OR was defined as at least a PR (PR+CR). No
response (NR) was defined as failure in any lineage. These definitions
have been previously reported [17]. Response time
referred to the time from IST to the first objective response.