Table 2.
Study treatment until further progression or end of study, and treatment at first LM progression in the ITT population
| Control Group (N = 37) | Experimental Group (N = 36) | |
|---|---|---|
| Intrathecal liposomal cytarabine until LM progression or end of study | ||
| Administration of liposomal cytarabine, n (%) | 1 (3%)1 | 36 (100%) |
| Duration of treatment, months, median (range) | 11.3 | 3.1 (0.4; 31.4) |
| Number of injections: median (range) | 14 | 5 (1–20) |
| Lumbar route only | 0 | 24 (67%) |
| Ventricular route | 1 | 12 (33%) |
| Time from randomization to first injection, days (range) | 0 | 0 (0; 13) |
| Systemic treatment, n (%)1until LM progression or end of study | 36 (97%) | 33 (92%) |
| Duration of treatment, months, median (range) | 2.4 (0.6; 44.8) | 3.6 (0.7; 18.2) |
| Type of systemic treatment (sometimes in combination) | ||
| Chemotherapy2 | 33 | 33 |
| Hormonal therapy | 1 | 1 |
| HER-2 targeted treatment | 7 | 2 |
| Other targeted treatment | 3 | 1 |
| Focal CNS radiotherapy, n (%) until LM progression or end of study(sometimes in combination)3 | 6 (16%) | 3 (8%) |
| Brain | 6 | 2 |
| Spine | 1 | 1 |
| Treatment at further LM progression, n (%)(possibly associated) | 30 (81%) | 23 (64%) |
| Intrathecal therapy | 15 | 2 |
| Focal radiotherapy | 1 | 2 |
| Systemic therapy | 19 | 10 |
1 Two patients in the control group and 4 patients in the experimental group had a major protocol violation. They are excluded from the per protocol population (see Fig. 1).
2 Systemic pharmacotherapy at study entry is detailed in Supplementary Table 9.
3 Six patients in the control group received focal radiotherapy: posterior fossa (n = 3), meningeal nodule at brain level (n = 1), cauda equina (n = 1), meningeal nodule at brain level and cauda equina (n = 1); 3 patients in the experimental group received focal radiotherapy: posterior fossa (n = 1), meningeal nodule at brain level (n = 1), cauda equina (n = 1).