Demographic and baseline characteristics
| . | R/R SAA n = 14 . | Treatment-naïve SAA n = 37 . | All patients N = 51 . |
|---|---|---|---|
| Age, median (range), y | 11.0 (4.0-17.0) | 10.0 (2.0-17.0) | 10.0 (2.0-17.0) |
| Sex, male, n (%) | 8 (57.1) | 20 (54.1) | 28 (54.9) |
| Race, n (%) | |||
| White | 6 (42.9) | 24 (64.9) | 30 (58.8) |
| Black | 3 (21.4) | 4 (10.8) | 7 (13.7) |
| Asian | 4 (28.6) | 9 (24.3) | 13 (25.5) |
| Unknown | 1 (7.1) | 0 | 1 (2.0) |
| BMI, median (range), kg/m2 | 17.5 (14.2-32.5) | 19.3 (11.9-34.6) | 17.9 (11.9-34.6) |
| Time since the first diagnosis, median (range), mo | 14.7 (3.1-105.4) | 1.2 (0.5-8.5) | 2.2 (0.5-105.4) |
| Performance status at baseline, median (min-max) | |||
| Lansky Play performance scale | 90 (80-100)∗ | 90 (70-100)† | 90 (70-100)‡ |
| Karnofsky performance scale | 80 (80-80)§ | 80 (80-100)|| | 80 (80-100)¶ |
| Baseline hematologic counts, median (range)# | |||
| Platelets, ×109/L | 31.5 (13-91) | 38.0 (4-126) | 35.0 (4-126) |
| Hemoglobin, g/dL | 9.3 (6.6-12.8) | 8.9 (5.7-11.7) | 8.9 (5.7-12.8) |
| Neutrophils, ×109/L | 0.6 (0-4.6) | 0.2 (0-2.3) | 0.2 (0-4.6) |
| Reticulocyte, ×109/L | 26.2 (0-107) | 12.4 (2-78) | 14.2 (0-107) |
| Baseline RBC transfusion dependent, n (%) | 10 (71.4) | 32 (86.5) | 42 (82.4) |
| Baseline platelet transfusion dependent, n (%) | 10 (71.4) | 33 (89.2) | 43 (84.3) |
| . | R/R SAA n = 14 . | Treatment-naïve SAA n = 37 . | All patients N = 51 . |
|---|---|---|---|
| Age, median (range), y | 11.0 (4.0-17.0) | 10.0 (2.0-17.0) | 10.0 (2.0-17.0) |
| Sex, male, n (%) | 8 (57.1) | 20 (54.1) | 28 (54.9) |
| Race, n (%) | |||
| White | 6 (42.9) | 24 (64.9) | 30 (58.8) |
| Black | 3 (21.4) | 4 (10.8) | 7 (13.7) |
| Asian | 4 (28.6) | 9 (24.3) | 13 (25.5) |
| Unknown | 1 (7.1) | 0 | 1 (2.0) |
| BMI, median (range), kg/m2 | 17.5 (14.2-32.5) | 19.3 (11.9-34.6) | 17.9 (11.9-34.6) |
| Time since the first diagnosis, median (range), mo | 14.7 (3.1-105.4) | 1.2 (0.5-8.5) | 2.2 (0.5-105.4) |
| Performance status at baseline, median (min-max) | |||
| Lansky Play performance scale | 90 (80-100)∗ | 90 (70-100)† | 90 (70-100)‡ |
| Karnofsky performance scale | 80 (80-80)§ | 80 (80-100)|| | 80 (80-100)¶ |
| Baseline hematologic counts, median (range)# | |||
| Platelets, ×109/L | 31.5 (13-91) | 38.0 (4-126) | 35.0 (4-126) |
| Hemoglobin, g/dL | 9.3 (6.6-12.8) | 8.9 (5.7-11.7) | 8.9 (5.7-12.8) |
| Neutrophils, ×109/L | 0.6 (0-4.6) | 0.2 (0-2.3) | 0.2 (0-4.6) |
| Reticulocyte, ×109/L | 26.2 (0-107) | 12.4 (2-78) | 14.2 (0-107) |
| Baseline RBC transfusion dependent, n (%) | 10 (71.4) | 32 (86.5) | 42 (82.4) |
| Baseline platelet transfusion dependent, n (%) | 10 (71.4) | 33 (89.2) | 43 (84.3) |