TABLE 2.
Patient characteristics
| Variables | All enrollments (N = 278) |
First enrollment (N = 213) |
First enrollment |
|
|---|---|---|---|---|
| MIBG protocols (N = 114) |
Non-MIBG protocols (N = 99) |
|||
| Age at diagnosis of HR-NB | ||||
| <18 months | 7(3%) | 7(3%) | 2 (2%) | 5(5%) |
| 18 months to 2 years | 51 (18%) | 39 (18%) | 23 (20%) | 16(16%) |
| 3–5 years | 119(43%) | 92 (43%) | 50 (44%) | 42 (42%) |
| 6–9 years | 52 (19%) | 37(17%) | 22(19%) | 15 (15%) |
| 10–20 years | 45 (16%) | 35 (16%) | 16(14%) | 19(19%) |
| ≥21 years | 4(1%) | 3 (1%) | 1 (1%) | 2 (2%) |
| Age at start of treatment (years) | ||||
| <3 | 9 (3%) | 9 (4%) | 6 (5%) | 3(3%) |
| 3–5 | 85 (31%) | 70 (33%) | 43 (38%) | 27(27%) |
| 6–9 | 88 (32%) | 66(31%) | 37 (32%) | 29 (29%) |
| 10–20 | 80 (29%) | 58 (27%) | 27 (24%) | 31(31%) |
| ≥21 | 16 (6%) | 10 (5%) | 1 (1%) | 9(9%) |
| Sex | ||||
| Female | 92 (33%) | 72 (34%) | 38 (33%) | 34 (34%) |
| Male | 186 (67%) | 141 (66%) | 76 (67%) | 65 (66%) |
| MYCN | ||||
| Amplified | 50 (24%) | 39 (24%) | 16(19%) | 23(31%) |
| Not amplified | 159(76%) | 121 (76%) | 69 (81%) | 52 (69%) |
| Unknown | 69 | 53 | 29 | 24 |
| Histopathology | ||||
| Favorable | 7(4%) | 7(5%) | 4(5%) | 3(4%) |
| Unfavorable | 178 (96%) | 135 (95%) | 69 (95%) | 66 (96%) |
| Unknown | 93 | 71 | 41 | 30 |
| Stage at initial diagnosis of NB | ||||
| 1 | 7(3%) | 5(2%) | 4(4%) | 1 (1%) |
| 2 | 12 (4%) | 7 (3%) | 4 (4%) | 3 (3%) |
| 3 | 16 (6%) | 11(5%) | 2 (2%) | 9(9%) |
| 4 | 242 (87%) | 189 (89%) | 103 (91%) | 86 (87%) |
| Unknown | 1 | 1 | 1 | 0 |
| PD prior to NANT enrollment | ||||
| Yes | 208 (75%) | 149 (70%) | 57 (50%) | 92 (93%) |
| No | 70 (25%) | 64 (30%) | 57 (50%) | 7(7%) |
| Baseline BM tumor content based on aspirates and biopsies | ||||
| Negative | 140 (51%) | 109(51%) | 58 (51%) | 51 (52%) |
| Positive | 137 (49%) | 103 (49%) | 56 (49%) | 47 (48%) |
| Unknown | 1 | 1 | 0 | 1 |
| Baseline BM tumor content based on biopsies only | ||||
| 0% | 151 (55%) | 117(55%) | 63 (55%) | 54 (55%) |
| >0 to ≤5% | 50 (18%) | 38(18%) | 21(18%) | 17(17%) |
| >5 to 10% | 6(2%) | 5 (2%) | 3 (3%) | 2 (2%) |
| <10% | 2 (<1%) | 2 (1%) | 0 | 2 (2%) |
| >5% | 1 (<1%) | 1 (<1%) | 1 (<1%) | 0 |
| >10 to 20% | 12 (4%) | 10 (5%) | 5(4%) | 5(5%) |
| >20 to 30% | 4(1%) | 3(1%) | 2 (2%) | 1 (1%) |
| >30% | 22 (8%) | 16 (8%) | 10 (9%) | 6(6%) |
| Positive NOS | 22 (8%) | 14 (7%) | 7(6%) | 7(7%) |
| No report/inadequate specimen | 7(3%) | 6(3%) | 2 (2%) | 4 (4%) |
| Not done | 1 | 1 | 0 | 1 |
| Baseline CT disease | ||||
| Negative/nonmeasurable | 135 (49%) | 110(52%) | 61 (54%) | 49 (49%) |
| Positive/measurable | 141(51%) | 103 (48%) | 53 (46%) | 50 (51%) |
| Unknown | 2 | 0 | 0 | 0 |
| Baseline MIBG scan | ||||
| Negative | 16 (6%) | 15 (7%) | 0 | 15(16%) |
| Positive | 255 (94%) | 194 (93%) | 114(100%) | 80 (84%) |
| Nonavid tumor | 7 | 4 | 0 | 4 |
| Baseline Curie score | ||||
| 0 | 16 (7%) | 15 (8%) | 0 | 15(19%) |
| 1–2 | 58 (25%) | 45 (25%) | 26 (26%) | 19 (24%) |
| 3–4 | 23(10%) | 17 (9%) | 10 (10%) | 7(9%) |
| 5–9 | 51 (22%) | 40 (22%) | 30 (30%) | 10(13%) |
| 10–19 | 60 (26%) | 47 (26%) | 27(27%) | 20 (25%) |
| 20–25 | 23(10%) | 16 (9%) | 8 (8%) | 8 (10%) |
| Nonavid tumor | 7 | 4 | 0 | 4 |
| Unknown | 40 | 29 | 13 | 16 |
| Median (range) | 6 (0, 25) | 6(0,25) | 7(1,25) | 4 (0, 24) |
| Best overall response to first NANT trial | ||||
| Complete response | 14 | 14 | 9 | 5 |
| Partialresponse | 16 | 14 | 13 | 1 |
| Mixed response | 27 | 24 | 22 | 2 |
| Stable disease | 110 | 81 | 43 | 38 |
| Progressive disease | 96 | 70 | 22 | 48 |
| Toxic death | 2 | 2 | 2 | 0 |
| Inevaluable | 13 | 8 | 3 | 5 |
| Follow-up (years) | ||||
| Median (range) | 4.6(0.32,11.6) | 4.6 (0.32,11.6) | 4.3(0.32,11.6) | 5.0 (3.4,6.9) |
HR-NB, high-risk neuroblastoma; MIBG, meta-iodobenzylguanidine; NB, neuroblastoma; PD, progressive disease; NANT, New Approaches to Neuroblastoma Therapy; BM, bone marrow; NOS, not otherwise specified.