Patient 1 2 3 4 5 Age/sex 5/M 7/F 15/M 13/M 16/M Current Age 17 19 27 - - Dx/HDCY (months)* 18 9 13 4 10 Follow-up (months) 162 153 157 1 3 G-CSF doses;5 mg/Kg/day 28 14 10 7 20 Days in the hospital 146 58 31 8 60 Packed red cells 33 15 5 2 8 Platelet concentrates 308 14 4 8 172 ANC>0.5×109/L† +126 +52 +55 - +65 PC>20×109/L ‡ +126 +53 +90 - - Current HB g/dL 8.4 15.4 16.2 - - Current WBC ´109/L 1.56 5.77 6.25 - - Current ANC´109/L 0.97 3.50 3.31 - - Current PC´109/L 49 172 181 - - Reticulocytes ´109/L 15 81 90 - - BMA MDS M N - - BM cellularity (biopsy) MDS↑ 60% 60% - -

*= Months from diagnosis of SAA to treatment with HDCY. †= Days from HDCY to an ANC>500 109/L. ‡= Days from HDCY to a PC>20 ×109/L. BMA= Bone Marrow Aspirate MDS= Mielodysplasia

Table 1: Clinical and laboratory data from children receiving high-dose cyclophosphamide (HDCY) plus granulocyte-colony stimulating factor (G-CSF) therapy for severe acquired aplastic anemia (SAA). Survivors were followed for 12 years.