Table 1.
Cohort characteristics
| Category | ||
|---|---|---|
| Discovery cohort (n = 36) | Pooled discovery and validation cohorts (n = 64) | |
| Mean (±SD) age at first imaging diagnosis, y | 62.07 (±10.01) | 61.27 (±10.75) |
| Sex | ||
| Male | 21 (58) | 35 (55) |
| Female | 15 (42) | 29 (45) |
| Diagnosis | ||
| Symptoms* | ||
| Altered mental status or confusion | 14 (39) | 25 (40) |
| Imbalance, dizziness, or vertigo | 9 (25) | 21 (33) |
| Weakness | 9 (25) | 13 (21) |
| Language difficulties, dysarthria, or aphasia | 10 (28) | 19 (30) |
| Other | 26 (72) | 51 (81) |
| Tumor focality | ||
| Single | 18 (50) | 34 (53) |
| Multiple | 18 (50) | 29 (45) |
| NR | 0 (0) | 1 (2) |
| Tumor location | ||
| Infratentorial | 3 (8) | 6 (9) |
| Supratentorial | 29 (81) | 48 (75) |
| Both | 4 (11) | 8 (13) |
| Midbrain | 0 (0) | 1 (2) |
| NR | 0 (0) | 1 (2) |
| Ocular involvement | ||
| Positive or suspicious | 4 (11) | 7 (10.9) |
| Negative | 31 (86) | 49 (76.6) |
| NR | 1 (3) | 8 (12.5) |
| CSF involvement† | ||
| Positive or suspicious | 8 (22) | 14 (22) |
| Negative | 18 (50) | 27 (42) |
| NR | 10 (28) | 23 (36) |
| Systemic imaging | ||
| Positive or suspicious | 1 (3) | 2 (3) |
| Negative | 35 (97) | 55 (86) |
| NR | 0 (0) | 7 (11) |
| Bone marrow biopsy | ||
| Positive or suspicious | 2 (6) | 2 (3.1) |
| Negative | 10 (28) | 15 (23.4) |
| NR | 24 (67) | 47 (73.4) |
| Testicular ultrasound | ||
| Positive or suspicious | 0 (0) | 0 (0) |
| Negative | 9 (25) | 17 (27) |
| NR | 27 (75) | 47 (73) |
| Therapy | ||
| Initial induction regimen | ||
| Methotrexate, temozolomide, and rituximab | 23 (64) | 41 (64) |
| Methotrexate ± rituximab | 13 (36) | 23 (36) |
| Autologous stem-cell transplantation | ||
| Received | 8 (22) | 19 (30) |
| Did not receive | 28 (78) | 45 (70) |
| Response | ||
| Response to initial therapy | ||
| CR | 30 (83.3) | 57 (89) |
| PR | 3 (8.3) | 4 (6) |
| PD | 3 (8.3) | 3 (5) |
| Relapse | ||
| ≥1 | 14 (39) | 25 (39) |
| First in brain | 10 (71‡) | 15 (60‡) |
| First ocular or systemic | 4 (29‡) | 10 (40‡) |
| Multiple | 5 (14) | 8 (12.5) |
| Follow-up and survival, mo | ||
| Median follow-up | 56.2 | |
| Median PFS | 111 (95% CI, 35 to ∞) | |
| Median OS | NR | |
| Median TTCR | 4 (95% CI, 3-6) |
Data are n (%) unless otherwise indicated.
CI, confidence interval; CR, complete response; CSF, cerebrospinal fluid; NR, not reported/performed; OS, overall survival; PD, progressive or refractory disease; PFS, progression-free survival; PR, partial response; SD, standard deviation; TTCR, time to complete response.
*
Symptoms not reported for 1 validation patient.
†
If evaluated within 1 month of diagnosis.
‡
Percentage of patients with specific location of first relapse of all patients who relapsed at least once.