Table 1.
Patient demographics and baseline characteristics
| Characteristic | 320 mg q.d. N = 20 | 480 mg q.d. N = 7 | 480 mg q.d., fasted N = 17 | All N = 44 |
|---|---|---|---|---|
| Sex, male, n (%) | 14 (70.0) | 4 (57.1) | 6 (35.3) | 24 (54.5) |
| Age, median, y (range) | 59.5 (41–86) | 54.0 (42–75) | 65.0 (29–85) | 60.0 (29–86) |
| Karnofsky performance status, n (%) | ||||
| <70 | 0 | 0 | 0 | 0 |
| 70 | 8 (40.0) | 3 (42.9) | 9 (52.9) | 20 (45.5) |
| 80 | 2 (10.0) | 0 (0.0) | 2 (11.8) | 4 (9.1) |
| 90 | 6 (30.0) | 2 (28.6) | 3 (17.6) | 11 (25.0) |
| 100 | 4 (20.0) | 2 (28.6) | 3 (17.6) | 9 (20.5) |
| Median (range) | 85.0 (70–100) | 90.0 (70–100) | 70.0 (70–100) | 80.0 (70–100) |
| Number of previous lines of treatment, n (%) | ||||
| 1 | 9 (45.0) | 2 (28.6) | 7 (41.2) | 18 (40.9) |
| 2–3 | 5 (25.0) | 3 (42.9) | 8 (47.1) | 16 (36.4) |
| ≥4 | 6 (30.0) | 2 (28.6) | 2 (11.8) | 10 (22.7) |
| Median (range) | 2.0 (1–6) | 2.0 (1–14) | 2.0 (1–5) | 2.0 (1–14) |
| Previous methotrexate | 20 (100.0) | 7 (100.0) | 17 (100.0) | 44 (100.0) |
| Previous rituximab | 13 (65.0) | 3 (42.9) | 10 (58.8) | 26 (59.1) |
| Previous whole-brain radiotherapy | 14 (70.0) | 5 (71.4) | 10 (58.8) | 29 (65.9) |
| Previous HCT-ASCT | 2 (10.0) | 1 (14.3) | 4 (37.8) | 7 (15.9) |
| Disease status, n (%) | ||||
| Relapsea | 17 (85.0) | 2 (28.6) | 14 (82.4) | 33 (75.0) |
| Refractoryb | 3 (15.0) | 3 (42.9) | 3 (17.6) | 9 (20.5) |
| Unknown | 0 | 2 (28.6) | 0 | 2 (4.5) |
| CNS involvement, n (%) | ||||
| CSF | ||||
| Positive | 1 (5.0) | 1 (14.3) | 7 (41.2) | 9 (20.5) |
| Negative | 19 (95.0) | 6 (85.7) | 10 (58.8) | 35 (79.5) |
| IOL | ||||
| Positive | 2 (10.0) | 0 | 1 (5.9) | 3 (6.8) |
| Minor RPE abnormality | 5 (25.0) | 0 | 1 (5.9) | 6 (13.6) |
| Negative | 13 (65.0) | 7 (100.0) | 15 (88.2) | 35 (79.5) |
| GCB subtype, n (%) | ||||
| GCB | 7 (35.0) | 1 (14.3) | 5 (29.4) | 13 (29.5) |
| Non-GCB | 13 (65.0) | 6 (85.7) | 12 (70.6) | 31 (70.5) |
| Oncogenic mutation, n (%) | ||||
| CARD11 | 3 (15.0) | 2 (28.6) | 12 (70.6) | 17 (38.6) |
| MYD88 | 15 (75.0) | 6 (85.7) | 11 (64.7) | 32 (72.7) |
| CD79B | 10 (50.0) | 0 | 8 (47.1) | 18 (40.9) |
| Sum of the products of the greatest diameters at target lesion, mm2, n (%) | ||||
| <400 | 13 (65.0) | 3 (42.9) | 7 (41.2) | 23 (52.3) |
| ≥400 | 7 (35.0) | 4 (57.1) | 10 (58.8) | 21 (47.7) |
| Median, mm2 (range) | 233.66 (56.3–2031.4) | 514.99 (77.1– 4020.5) | 618.58 (86.5–2047.7) | 385.84 (56.3–4020.5) |
Abbreviations: HCT-ASCT, high-dose chemotherapy followed by autologous stem cell transplantation; IOL, intraocular lymphoma; RPE, retinal pigment epithelium.
aA disease that responded to the last therapy (CR, CRu, or PR) but progressed afterward was defined as relapsed PCNSL.
bA disease that did not respond to the last therapy (stable or progressive disease) was defined as refractory PCNSL.