Efficacy of thalidomide for CGD-IBD in the trial
| Category | Definition | Period | Group | Categorization | Frequency | Achievement rate | 95% CIs (lower, upper limits) | Difference in achievement rate | 95% CIs of the difference (lower, upper limits) | Achievement rate ratio | 95% CIs of the ratio (lower, upper limits) |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary endpoint | PUCAI decrease ≥20 points or score <10 | Blinded phase | Thalidomide (n = 3) | Achieved | 1 | 0.33 | 0.01, 0.91 | 0.33 | −0.37, 0.91 | ∞ | 0.13, ∞ |
| Not achieved | 2 | ||||||||||
| Placebo (n = 5) | Achieved | 0 | 0 | 0, 0.52 | |||||||
| Not achieved | 5 | ||||||||||
| Extension phase | All (n = 8) | Achieved | 5 | 0.63 | 0.25, 0.92 | – | – | – | – | ||
| Not achieved | 3 | ||||||||||
| Secondary endpoint | PUCAI <10 | Blinded phase | Thalidomide (n = 3) | Achieved | 0 | 0 | 0, 0.71 | – | – | – | – |
| Not achieved | 3 | ||||||||||
| Placebo (n = 5) | Achieved | 0 | 0 | 0, 0.52 | |||||||
| Not achieved | 5 | ||||||||||
| Extension phase | All (n = 8) | Achieved | 5 | 0.63 | 0.25, 0.92 | – | – | – | – | ||
| Not achieved | 3 | ||||||||||
| PGA with ≥1 grade improvement | Blinded phase | Thalidomide (n = 3) | Achieved | 1 | 0.33 | 0.01, 0.91 | 0.13 | −0.54, 0.77 | – | – | |
| Not achieved | 2 | ||||||||||
| Placebo (n = 5) | Achieved | 1 | 0.20 | 0.01, 0.72 | |||||||
| Not achieved | 4 | ||||||||||
| Extension phase | All (n = 8) | Achieved | 5 | 0.63 | 0.25, 0.92 | – | – | – | – | ||
| Not achieved | 3 |
| Category | Definition | Period | Group | Categorization | Frequency | Achievement rate | 95% CIs (lower, upper limits) | Difference in achievement rate | 95% CIs of the difference (lower, upper limits) | Achievement rate ratio | 95% CIs of the ratio (lower, upper limits) |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary endpoint | PUCAI decrease ≥20 points or score <10 | Blinded phase | Thalidomide ( | Achieved | 1 | 0.33 | 0.01, 0.91 | 0.33 | −0.37, 0.91 | ∞ | 0.13, ∞ |
| Not achieved | 2 | ||||||||||
| Placebo ( | Achieved | 0 | 0 | 0, 0.52 | |||||||
| Not achieved | 5 | ||||||||||
| Extension phase | All ( | Achieved | 5 | 0.63 | 0.25, 0.92 | – | – | – | – | ||
| Not achieved | 3 | ||||||||||
| Secondary endpoint | PUCAI <10 | Blinded phase | Thalidomide ( | Achieved | 0 | 0 | 0, 0.71 | – | – | – | – |
| Not achieved | 3 | ||||||||||
| Placebo ( | Achieved | 0 | 0 | 0, 0.52 | |||||||
| Not achieved | 5 | ||||||||||
| Extension phase | All ( | Achieved | 5 | 0.63 | 0.25, 0.92 | – | – | – | – | ||
| Not achieved | 3 | ||||||||||
| PGA with ≥1 grade improvement | Blinded phase | Thalidomide ( | Achieved | 1 | 0.33 | 0.01, 0.91 | 0.13 | −0.54, 0.77 | – | – | |
| Not achieved | 2 | ||||||||||
| Placebo ( | Achieved | 1 | 0.20 | 0.01, 0.72 | |||||||
| Not achieved | 4 | ||||||||||
| Extension phase | All ( | Achieved | 5 | 0.63 | 0.25, 0.92 | – | – | – | – | ||
| Not achieved | 3 |
PUCAI, Pediatric Ulcerative Colitis Activity Index; PGA, Physician Global Assessment, ∞, infinity.
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