Table 1.

Patient characteristics

	Dose escalation (A1; n=43)	Confirmed genetic alterations (A2; n=25)	Neuroblastoma (A3; n=11)
Age (years)
Median (range)	11·5 (1·9–20·9)	8·4 (1·1–21·4)	7·9 (3·5–14·6)
Sex
Male	24 (56%)	12 (48%)	3 (27%)
Female	19 (44%)	13 (52%)	8 (73%)
Previous chemotherapy regimens
Median (range)	2 (0–8)	1 (0–11)	1 (1–8)
Previous treatment
Radiation therapy	23 (53%)	7 (28%)	9 (82%)
Bone marrow transplant	11 (26%)	3 (12%)	6 (55%)
Diagnosis
Solid tumours
Ewing’s sarcoma	4 (9%)	0	0
Inflammatory myofibroblastic tumour	2 (5%)	5 (20%)	0
Osteosarcoma	7 (16%)	0	0
Neuroblastoma	15 (35%)	8 (32%)	11 (100%)
Non-small-cell lung	0	3 (12%)	0
Other*	5 (12%)	0	0
Rhabdomyosarcoma	3 (7%)	0	0
Soft-tissue sarcoma	2 (5%)	0	0
Lymphomas
Anaplastic large-cell lymphoma	0	9 (36%)	0
CNS tumours
Glioma	4 (9%)	0	0
Ependymoma	1 (2%)	0	0
Performance status†
100	26 (60%)	9 (36%)	5 (45%)
90	9 (21%)	7 (28%)	4 (36%)
80	4 (9%)	4 (16%)	1 (9%)
70	2 (5%)	4 (16%)	0
60	2 (5%)	1 (4%)	1 (9%)

Data are number of patients (%) or median (range). Part A1=dose escalation. Part A2=confirmed ALK translocation, mutation, or amplification. Part A3=neuroblastoma.

^*Hepatocellular carcinoma (n=2); Wilms’ tumour (n=1); malignant schwannoma (n=1); fusiform malignancy (n=1).

^†Lansky ≥16 years or Karnofsky >16 years.