Patient demographics and baseline characteristics
| . | 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 . |
|---|---|---|---|---|
| Median age (range), y | 14.3 (12.2-17.4) | 7.3 (6.1-8.8) | 3.6 (2.3-5.8) | 7.6 (2.3-17.4) |
| Male, n (%) | 13 (72.2) | 5 (41.7) | 10 (66.7) | 28 (62.2) |
| Race, n (%) | ||||
| White | 11 (61.1) | 5 (41.7) | 4 (26.7) | 20 (44.4) |
| Asian | 3 (16.7) | 3 (25.0) | 5 (33.3) | 11 (24.4) |
| Missing | 4 (22.2) | 4 (33.3) | 6 (40.0) | 14 (31.1) |
| Median weight (range), kg | 44.5 (36.9-85.5) | 22.4 (17.8-27.6) | 14.5 (9.0-22.5) | 23.8 (9.0-85.5) |
| Median BMI (range), kg/m2 | 18.6 (15.4-28.2) | 16.1 (11.9-19.1) | 16.7 (12.8-18.9) | 17.4 (11.9-28.2) |
| Steroid refractory, n (%) | 15 (83.3) | 6 (50.0) | 11 (73.3) | 32 (71.1) |
| Treatment-naïve patients, n (%) | 3 (16.7) | 6 (50.0) | 4 (26.7) | 13 (28.9) |
| aGVHD grade at baseline, n (%) | ||||
| Grade 2 | 12 (66.7) | 8 (66.7) | 9 (60.0) | 29 (64.4) |
| Grade 3 | 5 (27.8) | 4 (33.3) | 3 (20.0) | 12 (26.7) |
| Grade 4 | 1 (5.6) | 0 | 3 (20.0) | 4 (8.9) |
| aGVHD organ involvement at baseline, n (%) | ||||
| Skin | 13 (72.2) | 9 (75.0) | 12 (80.0) | 34 (75.6) |
| Liver | 2 (11.1) | 0 | 1 (6.7) | 3 (6.7) |
| Upper GI | 3 (16.7) | 5 (41.7) | 2 (13.3) | 10 (22.2) |
| Lower GI | 6 (33.3) | 5 (41.7) | 7 (46.7) | 18 (40.0) |
| . | 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 . |
|---|---|---|---|---|
| Median age (range), y | 14.3 (12.2-17.4) | 7.3 (6.1-8.8) | 3.6 (2.3-5.8) | 7.6 (2.3-17.4) |
| Male, n (%) | 13 (72.2) | 5 (41.7) | 10 (66.7) | 28 (62.2) |
| Race, n (%) | ||||
| White | 11 (61.1) | 5 (41.7) | 4 (26.7) | 20 (44.4) |
| Asian | 3 (16.7) | 3 (25.0) | 5 (33.3) | 11 (24.4) |
| Missing | 4 (22.2) | 4 (33.3) | 6 (40.0) | 14 (31.1) |
| Median weight (range), kg | 44.5 (36.9-85.5) | 22.4 (17.8-27.6) | 14.5 (9.0-22.5) | 23.8 (9.0-85.5) |
| Median BMI (range), kg/m2 | 18.6 (15.4-28.2) | 16.1 (11.9-19.1) | 16.7 (12.8-18.9) | 17.4 (11.9-28.2) |
| Steroid refractory, n (%) | 15 (83.3) | 6 (50.0) | 11 (73.3) | 32 (71.1) |
| Treatment-naïve patients, n (%) | 3 (16.7) | 6 (50.0) | 4 (26.7) | 13 (28.9) |
| aGVHD grade at baseline, n (%) | ||||
| Grade 2 | 12 (66.7) | 8 (66.7) | 9 (60.0) | 29 (64.4) |
| Grade 3 | 5 (27.8) | 4 (33.3) | 3 (20.0) | 12 (26.7) |
| Grade 4 | 1 (5.6) | 0 | 3 (20.0) | 4 (8.9) |
| aGVHD organ involvement at baseline, n (%) | ||||
| Skin | 13 (72.2) | 9 (75.0) | 12 (80.0) | 34 (75.6) |
| Liver | 2 (11.1) | 0 | 1 (6.7) | 3 (6.7) |
| Upper GI | 3 (16.7) | 5 (41.7) | 2 (13.3) | 10 (22.2) |
| Lower GI | 6 (33.3) | 5 (41.7) | 7 (46.7) | 18 (40.0) |
Full analysis set. The last available assessment on, or before, the date of start of study treatment is defined as baseline assessment.
BMI, body mass index.