Table 1.
Baseline patient characteristics
| Characteristic | Overall, n = 49 | Cohort A1, n = 25 | Cohort A2, n = 15 | Cohort B2, n = 9 |
|---|---|---|---|---|
| Gender, no. (%) | ||||
| Male | 24 (49%) | 12 (48%) | 6 (40%) | 6 (67%) |
| Female | 25 (51%) | 13 (52%) | 9 (60%) | 3 (33%) |
| Years of age at study entry | ||||
| Median (range) | 9.08 (2.51, 50.45) | 6.28 (2.51, 17.18) | 24.83 (15.24, 50.45) | 6.67 (3.68, 12.69) |
| Time (months) from diagnosis to study enrollment | ||||
| Median time (range) | 23.46 (7.46, 253.42) | 24.47 (8.38, 179.4) | 31.11 (11.50, 253.4) | 16.95 (7.46, 43.89) |
| Race, no. (%) | ||||
| White | 37 (76%) | 18 (72%) | 13 (87%) | 6 (67%) |
| Black | 2 (4%) | 2 (8%) | 0 (0%) | 0 (0%) |
| Other | 1 (2%) | 1 (4%) | 0 (0%) | 0 (0%) |
| Not specified | 9 (18%) | 4 (16%) | 2 (13%) | 3 (33%) |
| Disease status, no. (%) | ||||
| Relapsed | 40 (82%) | 22 (88%) | 12 (80%) | 6 (67%) |
| Refractory/persistent | 9 (18%) | 3 (12%) | 3 (20%) | 3 (33%) |
| MYCN status at diagnosis, no. (%) | ||||
| Amplified | 12 (24%) | 8 (32%) | 1 (7%) | 3 (33%) |
| Not amplified | 37 (76%) | 17 (68%) | 14 (93%) | 6 (67%) |
| Prior therapy received, no. (%) | ||||
| Myeloablative transplant (yes) | 29 (59%) | 16 (64%) | 7 (47%) | 6 (67%) |
| GD2 immunotherapy (yes) | 35 (71%) | 20 (80%) | 9 (60%) | 6 (67%) |
| Topotecan/cyclophosphamide (yes) | 34 (69%) | 19 (76%) | 9 (60%) | 6 (67%) |
| Disease status at baseline, no. (%) | ||||
| Soft tissue target lesion(s) present | 35 (71%) | 23 (92%) | 8 (53%) | 4 (44%) |
| Bone marrow involved | 17 (35%) | 8 (32%) | 5 (33%) | 4 (44%) |
| Bone involved | 38 (78%) | 17 (68%) | 13 (87%) | 8 (89%) |
| ALK aberration, no. (%) | ||||
| F1174 (C or L) | 25 (51%) | 12 (48%) | 8 (53%) | 5 (56%) |
| F1245 (Y or L) | 6 (12%) | 6 (24%) | 0 (0%) | 0 (0%) |
| R1275 (Q or L) | 16 (33%) | 6 (24%) | 6 (40%) | 4 (44%) |
| D1276_R1279>E | 1 (2%) | 0 (0%) | 1 (7%) | 0 (0%) |
| ALK amplification | 1 (2%) | 1 (4%) | 0 (0%) | 0 (0%) |
| Prior ALK inhibitor therapy, no. (%) | ||||
| ALK inhibitor naive | 29 (59%) | 16 (64%) | 10 (67%) | 3 (34%) |
| Crizotinib | 3 (6%) | 2 (8%) | 1 (7%) | 0 (0%) |
| Other ALK inhibitor* | 1 (2%) | 0 (0%) | 1 (7%) | 0 (0%) |
| Crizotinib + chemotherapy§ | 9 (18%) | 4 (16%) | 2 (13%) | 3 (33%) |
| Other ALK inhibitor + chemotherapy | 4 (8%) | 1 (4%) | 0 (0%) | 3 (33%) |
| More than one ALK inhibitor | 3 (6%) | 2 (8%) | 1 (7%) | 0 (0%) |
| Duration of ALK TKI therapy, range (months) | 8.39 (0.20, 41.60) | 8.51 (2.66, 19.68) | 20.11 (6.64, 41.59) | 1.30 (0.20, 11.04) |
| Tumor tissue used for ALK testing, no. (%) | ||||
| Diagnosis | 9 (18%) | 4 (16%) | 2 (13%) | 3 (33%) |
| Second look surgery | 5 (10%) | 2 (8%) | 2 (13%) | 1 (11%) |
| Relapsed/progression | 31 (63%) | 16 (64%) | 10 (67%) | 5 (56%) |
| Persistent/refractory | 4 (8%) | 3 (12%) | 1 (7%) | 0 (0%) |
§ Two patients had crizotinib alone and also with chemotherapy.
*Ceritinib, alectinib or ensartinib