Patient demographics and baseline characteristics
| Parameter | Thalidomide (n = 3) | Placebo (n = 5) | ||||||
|---|---|---|---|---|---|---|---|---|
| T1 | T2 | T3 | P1 | P2 | P3 | P4 | P5 | |
| Age, years | 6 | 25 | 1 | 1 | 28 | 28 | 8 | 1 |
| Sex | Male | Male | Male | Male | Male | Male | Male | Male |
| Genotype | CYBB | CYBB | CYBB | CYBB | CYBB | CYBB | NCF2 | CYBB |
| Weight, kg | 14.6 | 67.1 | 9.6 | 10.7 | 46.5 | 51.2 | 23.7 | 9.3 |
| PUCAI at screening | 45 | 40 | 35 | 40 | 35 | 35 | 30 | 40 |
| PUCAI at 0 wk | 15 | 45 | 25 | 50 | 35 | 40 | 35 | 25 |
| PGA at screening | Moderate | Moderate | Moderate | Moderate | Moderate | Moderate | Mild | Mild |
| PGA at 0 wk | Mild | Moderate | Mild | Moderate | Moderate | Moderate | Mild | Mild |
| Infection at initiation of trial | – | – | – | – | – | – | – | Thymic abscess |
| Concomitant drugs at 0 wk | TMP-SMX | Meropenem | Cefditoren pivoxil | TMP-SMX | TMP-SMX | TMP-SMX | TMP-SMX | Cefmetazole |
| Itraconazole | Itraconazole | Itraconazole | Fluconazole | Itraconazole | Itraconazole | Itraconazole | Micafungin | |
| Parameter | Thalidomide ( | Placebo ( | ||||||
|---|---|---|---|---|---|---|---|---|
| T1 | T2 | T3 | P1 | P2 | P3 | P4 | P5 | |
| Age, years | 6 | 25 | 1 | 1 | 28 | 28 | 8 | 1 |
| Sex | Male | Male | Male | Male | Male | Male | Male | Male |
| Genotype | ||||||||
| Weight, kg | 14.6 | 67.1 | 9.6 | 10.7 | 46.5 | 51.2 | 23.7 | 9.3 |
| PUCAI at screening | 45 | 40 | 35 | 40 | 35 | 35 | 30 | 40 |
| PUCAI at 0 wk | 15 | 45 | 25 | 50 | 35 | 40 | 35 | 25 |
| PGA at screening | Moderate | Moderate | Moderate | Moderate | Moderate | Moderate | Mild | Mild |
| PGA at 0 wk | Mild | Moderate | Mild | Moderate | Moderate | Moderate | Mild | Mild |
| Infection at initiation of trial | – | – | – | – | – | – | – | Thymic abscess |
| Concomitant drugs at 0 wk | TMP-SMX | Meropenem | Cefditoren pivoxil | TMP-SMX | TMP-SMX | TMP-SMX | TMP-SMX | Cefmetazole |
| Itraconazole | Itraconazole | Itraconazole | Fluconazole | Itraconazole | Itraconazole | Itraconazole | Micafungin | |
PUCAI, Pediatric Ulcerative Colitis Activity Index; PGA, Physician Global Assessment; TMP-SMX, trimethoprim/sulfamethoxazole.
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