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Long-term outcome of ATG & CSA ± G-CSF in SAA
Supplementary Figure S1A, B). In multivariate analysis including age, severity and randomization for G-CSF as variables, treatment with G-CSF was not associated with better survival [G-CSF; relative risk (RR) 0.91, 95% confi- dence interval (95% CI): 0.55-1.49; P=0.70]; the relative risk was increased for very SAA (RR 1.95, 95% CI 1.19- 3.21, P=0.008) and older age (reference age <20 years; 20- 39 years, RR 1.77, 95% CI: 0.48-6.67, P=0.40; age 40-59 years, RR 4.96, 95% CI: 1.48-16.65, P=0.009; age ≥60 years, RR 9.08, 95% CI 2.78-29.73, P<0001). EFS at 15 years according to age group was as follows: 27%±17% for patients aged <20 years, 28±16% for patients 20-39 years old, 30±14% for patients 40-59 years old, and 12±12% for patients 60 years or older (P=0.023) (Figure 2B). In multivariate analysis, age group was no longer sig-
A
nificantly different for EFS (Table 2), although a notable, non-significant trend remained for patients 60 years or older.
Relapse, non-response to immunosuppression and need for subsequent stem cell transplantation
We evaluated relapse, non-response to immunosup- pression and the need for either subsequent SCT or sub- sequent courses of IST. There was no difference between patients treated with or without G-CSF with respect to relapse and the need for second-line treatment: the cumu- lative incidence of relapse for patients responding at day 120 was 30±10% for the G-CSF group, and 25±10% for the non-G-CSF group (P=0.54) (Figure 3A). Forty patients needed a second-line therapy for relapse (n=17), refracto-
B
Figure 2. Overall survival and event-free survival according to age groups. (A, B) Overall survival (A) and event-free survival (B) of patients with severe aplastic anemia treated with horse antithymocyte globulin and cyclosporine with or without granulocyte colony-stimulating factor according to age groups at time of randomization: patients <20 years, patients 20-39 years, patients 40-59 years, patients 60 years or older. Events included relapse, non-response at day 120, subsequent stem cell transplantation, myelodysplastic syndrome/acute myeloid leukemia, solid cancer, paroxysmal nocturnal hemoglobinuria or death. G-CSF: granulocyte colony-stimu- lating factor.
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