Table 3 Original EBS trial data analysis of the composite blister and QoL outcome with the prioritized and non-prioritized split per treatment period. 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 | Treatment period 1 | ||||
| Â | Prioritized GPC | ||||
Blister | 30 (54%) | 3 (5%) | Â | 0.48 | Â |
QoL | 17 (30%) | 0 (0%) | Â | 0.30 | Â |
Total | 47 (84%) | 3 (5%) | 6 (11%) | 0.79 (0.21;0.96) | 0.0077 |
| Â | Non-prioritized GPC | ||||
Blister | 30 (53%) | 3 (5%) | 23 (41%) | 0.48 | 0.0662 |
QoL | 43 (77%) | 2 (4%) | 11 (20%) | 0.73 | 0.0076 |
Total | Â | Â | Â | 0.61 (0.18;0.84) | 0.0134 |
| Â | Treatment period 2 | ||||
| Â | Prioritized GPC | ||||
Blister | 18 (32%) | 5 (9%) | Â | 0.23 | Â |
QoL | 16 (29%) | 9 (16%) | Â | 0.13 | Â |
Total | 34 (61%) | 14 (25%) | 8 (14%) | 0.36 (-0.24;0.76) | 0.2368 |
| Â | Non-prioritized GPC | ||||
Blister | 18 (32%) | 5 (9%) | 33 (49%) | 0.23 | 0.0701 |
QoL | 36 (64%) | 11 (20%) | 9 (16%) | 0.45 | 0.0019 |
Total | Â | Â | Â | 0.34 (-0.05;0.64) | 0.1073 |