Table 2 Original EBS trial data analysis of the composite blister and QoL outcomes with the prioritized, non-prioritized and matched GPC. The blister outcome indicates if a subject has a 40% reduction in blisters. NTB = Net Treatment Benefit, CI = Confidence Interval
From: Composite endpoints, including patient reported outcomes, in rare diseases
| Â | # wins | #losses | #ties | NTB (95%CI) | p-value two-sided |
|---|---|---|---|---|---|
| Â | Prioritized GPC | ||||
Blister | 99 (44%) | 24 (11%) | Â | 0.33 | Â |
QoL | 72 (32%) | 14 (6%) | Â | 0.26 | Â |
Total | 171 (76%) | 38 (17%) | 16 (7%) | 0.59 (0.19;0.82) | 0.0051 |
| Â | Non-prioritized GPC | ||||
Blister | 99 (44%) | 24 (11%) | 102 (45%) | 0.33 | 0.0701 |
QoL | 162 (72%) | 22 (10%) | 41 (18%) | 0.62 | 0.0019 |
Total | Â | Â | Â | 0.48 (0.21;0.68) | 0.0022 |
| Â | Matched GPC | ||||
Blister | 5 (38%) | 1 (15%) | Â | 0.23 | Â |
QoL | 5 (38%) | 0 (0%) | Â | 0.20 | Â |
Total | 10 (77%) | 2 (15%) | 1 (8%) | 0.62 (0.09;0.88) | 0.0209 |