Table 2. Effectiveness endpoints in modified intention-to-treat population.
Figures are numbers of participants (percentage) unless stated otherwise.
| Low dose | High dose | Risk difference [95%CI] | p-value | HR* [95%CI] | p-value** | |
|---|---|---|---|---|---|---|
| n = 76 | n = 78 | |||||
| Primary composite outcome | 37 (48·7%) | 30 (38·5%) | 10·2% [-6·7; 27·1] | 0·264 | 0·72[0·45; 1·17] | 0·199 |
| Worsening toxicity1 | 31 (43·7%) | 27 (35·5%) | 8·1% [-9·0; 25·3] | 0·401 | ||
| Need for ventilation | 15 (19·7%) | 13 (16·7%) | 3·1% [-10·4; 16·6] | 0·776 | ||
| Death | 2 (2·6%) | 7 (9·0%) | -6·3% [-14·9; 2·2] | 0·167 | ||
| Number of vials, mean ± sd | 11·0 ± 7·9 | 12·5 ± 3·9 | 1·5 [-0·5; 3·5]*** | 0·142 | ||
| < 10 vials | 41 (53·9%) | 0 (0%) | ||||
| 10 to 15 vials | 17 (22·4%) | 70 (89·7%) | ||||
| > 15 vials | 18 (23·7%) | 8 (10·3%) |
1Seven patients had missing data for neurotoxicity score, 5 in the low dose group and 2 in the high dose group. All of these patients required ventilation so that a positive response to the primary composite outcome could be defined. It follows that no missing data remained for the primary composite outcome.
*adjusted for center;
**log-rank test stratified for center;
***mean difference [95%CI], p value from a Welch t test