Table 2.
A summary of the output from 5,000 simulated trial datasets for the expected treatment effect in PIMS (p0 = 0.10 for control arm (Na77), p1 = 0.04 for treatment arm (Na140))
| Variable | Description of the variable | Minimum | Q1 | Median | Mean | Q3 | Maximum |
|---|---|---|---|---|---|---|---|
| nevents0 | Number of events in the control group | 14 | 23 | 28 | 27 | 31 | 49 |
| nevents1 | Number of events in the treatment group | 1 | 9 | 11 | 11 | 14 | 24 |
| pevents0 | Proportion of events in the control group | 0.05 | 0.09 | 0.10 | 0.10 | 0.11 | 0.27 |
| pevents1 | Proportion of events in the treatment group | 0.01 | 0.03 | 0.04 | 0.04 | 0.05 | 0.08 |
| sample_size | Sample size | 129 | 584 | 584 | 545 | 584 | 584 |
| interim_time | Time at the interim (days) | 116 | 240 | 281 | 284 | 324 | 548 |
| interim_or | Odds ratio at the interim | 0.04 | 0.23 | 0.40 | 0.41 | 0.52 | 1.95 |
| interim_lci | Lower CI for OR at interim | 0.00 | 0.07 | 0.14 | 0.14 | 0.19 | 0.78 |
| interim_uci | Upper CI for OR at interim | 0.21 | 0.65 | 1.03 | 1.06 | 1.30 | 5.31 |
| interim_p | P-value for any difference between treatments at the interim analysis | 0.00 | 0.01 | 0.07 | 0.14 | 0.17 | 1.00 |
| interim_stop | Whether the trial would have stopped at the interim (based on decision criteria at interim) | 0.00 | 0.00 | 0.00 | 0.13 | 0.00 | 1.00 |
| final_or | Odds ratio at final analysis | 0.06 | 0.29 | 0.37 | 0.39 | 0.48 | 1.24 |
| final_lci | Lower CI for odds ratio | 0.01 | 0.13 | 0.18 | 0.19 | 0.24 | 0.65 |
| final_uci | Upper CI for odds ratio | 0.19 | 0.58 | 0.74 | 0.77 | 0.91 | 2.41 |
| final_p | P-value for treatment difference at final analysis | 0.00 | 0.00 | 0.01 | 0.04 | 0.03 | 1.00 |
| final_stop | Whether the trial is conclusive at final analysis | 0.00 | 1.00 | 1.00 | 0.80 | 1.00 | 1.00 |
| stop | Whether the trial was conclusive (at the interim or at the final analysis) | 0.00 | 1.00 | 1.00 | 0.80 | 1.00 | 1.00 |
| flipflop | The probability of trial flip-flopping | 0.000 | 0.000 | 0.000 | 0.001 | 0.000 | 1.000 |
Q1: 1st quartile; Q3: 3rd quartile. In bold are the quantities discussed in the manuscript. Namely, the average sample size, probability of trial stopping early (at interim), average of final odds ratio and confidence interval [CI] (lower CI boundary [LCI], upper CI boundary [UCI]), p-value, probability of trial success, probability of trial flipflopping (rounded to 3 decimal places due to small magnitude)