Table 1.
Demographics, clinical characteristics and outcome of patients according to consensus molecular grouping
| Characteristics (number with data available) | Entire cohort | Molecular Groups | P value | ||||
|---|---|---|---|---|---|---|---|
|
| |||||||
| PB-miRNA1 | PB-miRNA2 | PB-MYC/FOXR2 | PB-RB1 | PPTID | |||
| N=221 | N=96 | N=23 | N=34 | N=25 | N=43 | ||
|
| |||||||
| Age of diagnosis (N=188) median (range) – year(s) | 8.4 (0.0–64.0) | 8.5 (2.00–41.5) | 11.8 (1.33–31.5) | 1.4 (0.0–21.0) | 2.1 (0.10–3.94) | 33.0 (3.50–64.0) | <0.001 |
| <3 years | 42 (22%) | 5 (6%) | 1 (5%) | 19 (73%) | 17 (77%) | 0 | <0.001 |
| ≥3 – <18 years | 115 (61%) | 71 (86%) | 19 (86%) | 6 (23%) | 5 (23%) | 14 (40%) | |
| ≥18 years | 31 (17%) | 7 (8%) | 2 (9%) | 1 (4%) | 0 | 21 (60%) | |
| Sex (N=221) | 0.003 | ||||||
| Female | 113 (51%) | 59 (62%) | 9 (39%) | 8 (24%) | 13 (52%) | 24 (56%) | |
| Male | 108 (49%) | 37 (39%) | 14 (61%) | 26 (77%) | 12 (48%) | 19 (44%) | |
| Metastasis (N=138) | 0.01 | ||||||
| Yes | 54 (39%) | 28 (42%) | 3 (16%) | 9 (43%) | 11 (69%) | 3 (20%) | |
| No | 84 (61%) | 39 (58%) | 16 (84%) | 12 (57%) | 5 (31%) | 12 (80%) | |
| Histology (N=217) | -- | ||||||
| Pineoblastoma | 128 (59%) | 61 (65%) | 17 (74%) | 21 (62%) | 12 (48%) | 17 (42%) | |
| PNET | 58 (27%) | 32 (34%) | 5 (22%) | 10 (29%) | 7 (28%) | 4 (10%) | |
| Trilateral retinoblastoma | 6 (3%) | 0 | 0 | 0 | 6 (24%) | 0 | |
| Pineal anlage tumor | 2 (1%) | 0 | 0 | 2 (6%) | 0 | 0 | |
| PPTID | 21 (10%) | 1 (1%) | 1 (4%) | 1 (3%) | 0 | 18 (44%) | |
| Pineocytoma | 1 (1%) | 0 | 0 | 0 | 0 | 1 (2%) | |
| Central neurocytoma | 1 (1%) | 0 | 0 | 0 | 0 | 1 (2%) | |
| Surgery (N=134) | 0.64 | ||||||
| Gross/near-total resection | 63 (47%) | 27 (43%) | 9 (50%) | 11 (55%) | 6 (37.5%) | 10 (59%) | |
| Subtotal resection/biopsy | 71 (53%) | 36 (57%) | 9 (50%) | 9 (45%) | 10 (62.5%) | 7 (41%) | |
| Radiotherapy (N=122) | <0.001 | ||||||
| Craniospinal irradiation | 87 (71%) | 56 (88%) | 14 (88%) | 4 (22.2%) | 6 (54.5%) | 7 (54%) | |
| Focal radiotherapy | 11 (9%) | 3 (5%) | 1 (6%) | 4 (22.2%) | 0 | 3 (23%) | |
| No radiotherapy | 24 (20%) | 5 (8%) | 1 (6%) | 10 (55.6%) | 5 (45.5%) | 3 (23%) | |
| Chemotherapy (N=126) | <0.001 | ||||||
| High-dose | 58 (46%) | 36 (56%) | 8 (44%) | 5 (27.8%) | 4 (36.4%) | 5 (33%) | |
| Standard-dose | 57 (45%) | 28 (44%) | 7 (39%) | 11 (61.1%) | 7 (63.6%) | 4 (27%) | |
| No chemotherapy | 11 (9%) | 0 | 3 (17%) | 2 (11.1%) | 0 | 6 (40%) | |
|
Duration of follow-up median (range) – year(s) |
4.00 (0.00–17.0) | 4.88 (0.58–14.6) | 4.09 (0.50–10.1) | 1.33 (0.00–17.0) | 1.50 (0.20–13.7) | 4.75 (0.25–13.9) | <0.001 |
| Progression-free survival (PFS) | <0.001 | ||||||
| 2-year PFS (95%CI) | 66.7% (59.3–74.9) | 74.8% (65.1–86.0) | 94.7% (85.2–100) | 33.3% (18.9–58.7) | 38.5% (21.2–69.8) | 80.8% (63.4–100) | |
| 5-year PFS (95%CI) | 51.4% (43.5–60.8) | 56.7% (45.7–70.4) | 86.1% (69.5–100) | 16.7% (7.0–44.4) | 19.2% (7.1–52.2) | 80.8% (63.4–100) | |
| Overall survival (OS) | <0.001 | ||||||
| 2-year OS (95%CI) | 80.5% (74.3–87.3) | 92.4% (86.1–99.0) | 100% | 41.7% (26.0–66.9) | 55.9% (37.1–84.2) | 93.3% (81.5–100) | |
| 5-year OS (95%CI) | 62.2% (54.2–71.4) | 70.3% (59.6–82.9) | 100% | 23.8% (10.7–52.8) | 29.8% (14.0–63.6) | 86.2% (70.0–100) | |
CI, confidence interval; PNET, primitive neuroectodermal tumor; PPTID, pineal parenchymal tumor of intermediate differentiation