Summary of AEs
| . | Group 1 ≥12 y to <18 y n = 18 . | Group 2 ≥6 y to <12 y n = 12 . | Group 3 ≥2 y to <6 y n = 15 . | All patients N = 45 . | ||||
|---|---|---|---|---|---|---|---|---|
| All grades, n (%) . | Grade ≥3, n (%) . | All grades, n (%) . | Grade ≥3, n (%) . | All grades, n (%) . | Grade ≥3, n (%) . | All grades, n (%) . | Grade ≥3, n (%) . | |
| AEs | 18 (100) | 16 (88.9) | 12 (100) | 11 (91.7) | 15 (100) | 12 (80.0) | 45 (100) | 39 (86.7) |
| Treatment related | 10 (55.6) | 8 (44.4) | 7 (58.3) | 4 (33.3) | 6 (40.0) | 6 (40.0) | 23 (51.1) | 18 (40.0) |
| Most frequently (>4%) reported treatment-related AEs by PT | ||||||||
| Anemia | 1 (5.6) | 1 (5.6) | 4 (33.3) | 2 (16.7) | 4 (26.7) | 3 (20.0) | 9 (20.0) | 6 (13.3) |
| Neutrophil count decreased | 3 (16.7) | 3 (16.7) | 1 (8.3) | 1 (8.3) | 4 (26.7) | 4 (26.7) | 8 (17.8) | 8 (17.8) |
| White blood cell count decreased | 0 | 0 | 2 (16.7) | 2 (16.7) | 5 (33.3) | 4 (26.7) | 7 (15.6) | 6 (13.3) |
| Platelet count decreased | 0 | 0 | 2 (16.7) | 1 (8.3) | 4 (26.7) | 4 (26.7) | 6 (13.3) | 5 (11.1) |
| Neutropenia | 2 (11.1) | 2 (11.1) | 0 | 0 | 1 (6.7) | 1 (6.7) | 3 (6.7) | 3 (6.7) |
| Blood creatinine increased | 0 | 0 | 1 (8.3) | 0 | 1 (6.7) | 0 | 2 (4.4) | 0 |
| Constipation | 0 | 0 | 1 (8.3) | 0 | 1 (6.7) | 0 | 2 (4.4) | 0 |
| SAEs | 11 (61.1) | 10 (55.6) | 7 (58.3) | 6 (50.0) | 6 (40.0) | 4 (26.7) | 24 (53.3) | 20 (44.4) |
| Treatment related | 4 (22.2) | 4 (22.2) | 1 (8.3) | 1 (8.3) | 2 (13.3) | 1 (6.7) | 7 (15.6) | 6 (13.3) |
| Fatal SAEs | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Treatment related | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| AEs leading to discontinuation | 5 (27.8) | 4 (22.2) | 3 (25.0) | 3 (25.0) | 2 (13.3) | 2 (13.3) | 10 (22.2) | 9 (20.0) |
| Treatment related | 4 (22.2) | 3 (16.7) | 2 (16.7) | 2 (16.7) | 2 (13.3) | 2 (13.3) | 8 (17.8) | 7 (15.6) |
| AEs leading to dose adjustment/interruption | 11 (61.1) | 10 (55.6) | 5 (41.7) | 3 (25.0) | 7 (46.7) | 7 (46.7) | 23 (51.1) | 20 (44.4) |
| AEs requiring additional therapy | 16 (88.9) | 14 (77.8) | 12 (100) | 9 (75.0) | 15 (100) | 12 (80.0) | 43 (95.6) | 35 (77.8) |
| . | Group 1 ≥12 y to <18 y n = 18 . | Group 2 ≥6 y to <12 y n = 12 . | Group 3 ≥2 y to <6 y n = 15 . | All patients N = 45 . | ||||
|---|---|---|---|---|---|---|---|---|
| All grades, n (%) . | Grade ≥3, n (%) . | All grades, n (%) . | Grade ≥3, n (%) . | All grades, n (%) . | Grade ≥3, n (%) . | All grades, n (%) . | Grade ≥3, n (%) . | |
| AEs | 18 (100) | 16 (88.9) | 12 (100) | 11 (91.7) | 15 (100) | 12 (80.0) | 45 (100) | 39 (86.7) |
| Treatment related | 10 (55.6) | 8 (44.4) | 7 (58.3) | 4 (33.3) | 6 (40.0) | 6 (40.0) | 23 (51.1) | 18 (40.0) |
| Most frequently (>4%) reported treatment-related AEs by PT | ||||||||
| Anemia | 1 (5.6) | 1 (5.6) | 4 (33.3) | 2 (16.7) | 4 (26.7) | 3 (20.0) | 9 (20.0) | 6 (13.3) |
| Neutrophil count decreased | 3 (16.7) | 3 (16.7) | 1 (8.3) | 1 (8.3) | 4 (26.7) | 4 (26.7) | 8 (17.8) | 8 (17.8) |
| White blood cell count decreased | 0 | 0 | 2 (16.7) | 2 (16.7) | 5 (33.3) | 4 (26.7) | 7 (15.6) | 6 (13.3) |
| Platelet count decreased | 0 | 0 | 2 (16.7) | 1 (8.3) | 4 (26.7) | 4 (26.7) | 6 (13.3) | 5 (11.1) |
| Neutropenia | 2 (11.1) | 2 (11.1) | 0 | 0 | 1 (6.7) | 1 (6.7) | 3 (6.7) | 3 (6.7) |
| Blood creatinine increased | 0 | 0 | 1 (8.3) | 0 | 1 (6.7) | 0 | 2 (4.4) | 0 |
| Constipation | 0 | 0 | 1 (8.3) | 0 | 1 (6.7) | 0 | 2 (4.4) | 0 |
| SAEs | 11 (61.1) | 10 (55.6) | 7 (58.3) | 6 (50.0) | 6 (40.0) | 4 (26.7) | 24 (53.3) | 20 (44.4) |
| Treatment related | 4 (22.2) | 4 (22.2) | 1 (8.3) | 1 (8.3) | 2 (13.3) | 1 (6.7) | 7 (15.6) | 6 (13.3) |
| Fatal SAEs | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Treatment related | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| AEs leading to discontinuation | 5 (27.8) | 4 (22.2) | 3 (25.0) | 3 (25.0) | 2 (13.3) | 2 (13.3) | 10 (22.2) | 9 (20.0) |
| Treatment related | 4 (22.2) | 3 (16.7) | 2 (16.7) | 2 (16.7) | 2 (13.3) | 2 (13.3) | 8 (17.8) | 7 (15.6) |
| AEs leading to dose adjustment/interruption | 11 (61.1) | 10 (55.6) | 5 (41.7) | 3 (25.0) | 7 (46.7) | 7 (46.7) | 23 (51.1) | 20 (44.4) |
| AEs requiring additional therapy | 16 (88.9) | 14 (77.8) | 12 (100) | 9 (75.0) | 15 (100) | 12 (80.0) | 43 (95.6) | 35 (77.8) |
Safety Set. A patient with multiple severity grades for an AE is only counted under the maximum grade. AEs occurring during treatment or within 30 days of the last study medication are summarized. AEs were coded according to MedDRA version 25.1 and common toxicity criteria for adverse events version 4.03.
PT, preferred term.