Table 1.
Baseline Characteristics of the Study Patients
| Children (3 ≥ Age < 19 years) | Adults (≥19 years) | |||||
|---|---|---|---|---|---|---|
| Group 1 (20 mg/m2 q 12 h) |
Group 2 (25 mg/m2 q 12 h) |
Subtotal | Group 3 (50 mg q 12 h) |
Group 4 (25 mg/m2 q 12 h) |
Subtotal | |
| Patients enrolled—no. | 30 | 30 | 60 | 10 | 20 | 30 |
| NF1 | 29 | 29 | 58 | 10 | 18 | 28 |
| Mosaic NF1 | 1 | 3 | 4 | 1 | 2 | 3 |
| Sex—no. | ||||||
| Male | 15 | 20 | 35 | 6 | 14 | 20 |
| Female | 15 | 10 | 25 | 4 | 6 | 10 |
| Median age at enrollment (range)—year | 9.0 (4.0–18.0) | 8.5 (3.0–18.0) | 8.0 (3.0–18.0) | 34.0 (19.0–42.0) | 24.0 (19.0–47.0) | 26.5 (19.0–47.0) |
| Median performance status score (range) | 100 (70–100) | 100 (70–100) | 100 (70–100) | 90 (70–100) | 95 (70–100) | 90 (70–100) |
| Target location of plexiform neurofibroma—no. | ||||||
| abdomen | 2 | 1 | 3 | 2 | 2 | |
| ankle | 1 | 1 | ||||
| arm | 2 | 3 | 5 | |||
| arm, pelvis | 1 | 1 | ||||
| arm, pelvis, thigh | 1 | 1 | ||||
| arm, shoulder, back, pelvis | 1 | 1 | ||||
| buttock | 1 | 1 | ||||
| chest | 1 | 1 | 1 | 1 | ||
| chest wall | 1 | 1 | ||||
| chest, leg | 1 | 1 | ||||
| eye | 4 | 4 | ||||
| face | 1 | 1 | ||||
| face, chest | 1 | 1 | ||||
| face, paraspinal | 1 | 1 | ||||
| face, pelvis | 1 | 1 | ||||
| foot | 1 | 1 | ||||
| leg | 1 | 3 | 4 | 1 | 1 | 2 |
| liver | 1 | 1 | ||||
| neck | 6 | 9 | 15 | 4 | 1 | 5 |
| neck and pelvis | 1 | 1 | ||||
| neck, abdomen | 1 | 1 | ||||
| neck, back | 1 | 1 | ||||
| neck, chest | 1 | 1 | ||||
| neck, face | 1 | 1 | ||||
| neck, pelvis | 1 | 1 | 1 | 2 | 2 | |
| orbit | 1 | 1 | ||||
| paraspinal | 2 | 2 | 4 | 1 | 1 | |
| paraspinal, chest | 1 | 1 | ||||
| pelvis | 5 | 3 | 8 | 2 | 5 | 7 |
| pelvis, chest | 1 | 1 | ||||
| thoracic paraspinal | 1 | 1 | 1 | 1 | ||
| thoracic paraspinal, leg | 1 | 1 | ||||
| Median target plexiform neurofibroma volume at enrollment (range)—mL | 50.2 (3.4–922.6) | 70.6 (2.5–1137.5) | 60.7 (2.5–1137.5) | 71.2 (15.3–278.6) | 92.2 (5.7–546.8) | 84.5 (5.7–546.8) |
| Documented neurofibroma–related complication at baseline—no. (%) | ||||||
| Café-au-lait spot | 29 (96.7) | 30 (100) | 59 (98.3) | 10 (100.0) | 19 (95.0) | 29 (96.7) |
| Diffuse cutaneous neurofibroma | 14 (46.7) | 13 (43.3) | 27 (45.0) | 9 (90.0) | 14 (70.0) | 23 (76.7) |
| Mental retardation | 7 (23.3) | 7 (23.3) | 14 (23.3) | 3 (30.0) | 3 (15.0) | 6 (20.0) |
| Attention deficient hyperactivity disorder | 15 (50.0) | 12 (40.0) | 27 (45.0) | 2 (20.0) | 5 (25.0) | 7 (23.3) |
| Autism | 0 | 0 | 0 | 0 | 1 (5.0) | 1 (3.3) |
| Seizure | 1 (3.3) | 0 | 1 (1.7) | 0 | 0 | 0 |
| Hypertension | 0 | 2 (6.7) | 2 (3.3) | 2 (20.0) | 2 (10.0) | 4 (13.3) |
| Cardiac abnormality | 1 (3.3) | 0 | 1 (1.7) | 0 | 2 (10.0) | 2 (6.7) |
| Hearing abnormality | 3 (10.0) | 1 (3.3) | 4 (6.7) | 1 (10.0) | 3 (15.0) | 4 (13.3) |
| Optic pathway abnormality | 3 (10.0) | 3 (10.0) | 6 (10.0) | 0 | 0 | 0 |
| Dysplasia of bone | 12 (40.0) | 10 (33.3) | 22 (36.7) | 1 (10.0) | 10 (50.0) | 11 (36.7) |
| Median cycle at analysis (range) | 21 (4–26) | 22 (16–26) | 21 (4–26) | 26 | 26 (16–26) | 26 (16–26) |
NF1, neurofibromatosis type 1; no., number.