Patient characteristics at baseline
| Characteristic . | Study 1 (N = 43*) . | Study 2 (N = 40) . | P . |
|---|---|---|---|
| Age, y | |||
| Median | 45 | 50 | .399 |
| Range | 17-77 | 4-79 | |
| Age category, n (%) | |||
| <18 y | 2 (4.7) | 9 (22.5) | |
| 18-64 y | 27 (62.8) | 25 (62.5) | |
| ≥65 y | 14 (32.6) | 6 (15.0) | |
| Sex, n (%) | |||
| Female | 19 (44.2) | 19 (47.5) | .766 |
| Male | 24 (55.8) | 21 (52.5) | |
| Ethnicity, n (%) | |||
| Asian | 1 (2.3) | 5 (12.5) | |
| Black | 13 (30.2) | 4 (10.0) | |
| Hispanic | 9 (20.9) | 4 (10.0) | |
| White | 20 (46.5) | 27 (67.5) | |
| Other | 0 (0) | 0 (0) | |
| Previous treatments | |||
| No. of ISTs, average | 2.3 | 1.5 | <.001 |
| No. of ISTs, median (range) | 2 (1-4) | 1 (1-4) | |
| Time since last IST, median (range), mo | 9 (6-120) | 10 (6-68) | |
| Time since first IST, median (range), mo | 21 (7-189) | 13 (7-198) | |
| Laboratory parameters | |||
| Neutrophil count, ×109/L | |||
| Median | 0.57 | 0.70 | .202 |
| Range | 0.07-2.81 | 0.01-3.10 | |
| Reticulocyte count, ×109/L | |||
| Median | 24.0 | 32.3 | .224 |
| Range | 2.20-100.00 | 1.90-130.40 | |
| Platelet count, ×109/L | |||
| Median | 13.00 | 14.50 | .646 |
| Range | 5.00-65.00 | 2.00-53.00 | |
| Hemoglobin, g/dL | |||
| Median | 8.40 | 8.65 | .540 |
| Range | 6.00-13.30 | 5.90-12.40 | |
| Disease status, n (%)† | |||
| Severe AA | 36 (84) | 32 (80) | .778 |
| Super SAA | 6 (14) | 3 (9) | .485 |
| Transfusion dependency, n (%) | |||
| Platelets | 42 (98) | 30 (76.9) | .005 |
| Red cells | 40 (93) | 35 (89.7) | .703 |
| Characteristic . | Study 1 (N = 43*) . | Study 2 (N = 40) . | P . |
|---|---|---|---|
| Age, y | |||
| Median | 45 | 50 | .399 |
| Range | 17-77 | 4-79 | |
| Age category, n (%) | |||
| <18 y | 2 (4.7) | 9 (22.5) | |
| 18-64 y | 27 (62.8) | 25 (62.5) | |
| ≥65 y | 14 (32.6) | 6 (15.0) | |
| Sex, n (%) | |||
| Female | 19 (44.2) | 19 (47.5) | .766 |
| Male | 24 (55.8) | 21 (52.5) | |
| Ethnicity, n (%) | |||
| Asian | 1 (2.3) | 5 (12.5) | |
| Black | 13 (30.2) | 4 (10.0) | |
| Hispanic | 9 (20.9) | 4 (10.0) | |
| White | 20 (46.5) | 27 (67.5) | |
| Other | 0 (0) | 0 (0) | |
| Previous treatments | |||
| No. of ISTs, average | 2.3 | 1.5 | <.001 |
| No. of ISTs, median (range) | 2 (1-4) | 1 (1-4) | |
| Time since last IST, median (range), mo | 9 (6-120) | 10 (6-68) | |
| Time since first IST, median (range), mo | 21 (7-189) | 13 (7-198) | |
| Laboratory parameters | |||
| Neutrophil count, ×109/L | |||
| Median | 0.57 | 0.70 | .202 |
| Range | 0.07-2.81 | 0.01-3.10 | |
| Reticulocyte count, ×109/L | |||
| Median | 24.0 | 32.3 | .224 |
| Range | 2.20-100.00 | 1.90-130.40 | |
| Platelet count, ×109/L | |||
| Median | 13.00 | 14.50 | .646 |
| Range | 5.00-65.00 | 2.00-53.00 | |
| Hemoglobin, g/dL | |||
| Median | 8.40 | 8.65 | .540 |
| Range | 6.00-13.30 | 5.90-12.40 | |
| Disease status, n (%)† | |||
| Severe AA | 36 (84) | 32 (80) | .778 |
| Super SAA | 6 (14) | 3 (9) | .485 |
| Transfusion dependency, n (%) | |||
| Platelets | 42 (98) | 30 (76.9) | .005 |
| Red cells | 40 (93) | 35 (89.7) | .703 |
Total number of patients enrolled 44. One patient withdrew from study before the first dose of EPAG.
Disease status based on blood cell count criteria at entry; those with a super severe ANC (<0.2 × 109/L) also included in SAA category. Remainder of patients were qualified based on history of prior SAA and blood count/transfusion criteria as described in Methods.