Table 1.
Baseline Demographics and Physical Characteristics by UDCA Group: Primary Analysis Population
| UDCA Response Group | Total (N = 22) | P Value* (Overall) | P Value† | |||
|---|---|---|---|---|---|---|
| Null (n = 7) | Flare (n = 8) | Indeterminant (n = 7) | ||||
| Age at diagnosis (years) | ||||||
| n | 7 | 8 | 7 | 22 | 0.101 | |
| Mean (SD) | 9.7 (2.2) | 12.4 (4.4) | 13.9 (3.2) | 12.0 (3.7) | ||
| Age at start of phase I (years) | ||||||
| n | 7 | 8 | 7 | 22 | 0.157 | |
| Mean (SD) | 12.6 (2.1) | 13.4 (3.8) | 15.9 (3.3) | 13.9 (3.3) | ||
| Sex | ||||||
| Male | 5 (71.4%) | 6 (75.0%) | 6 (85.7%) | 17 (77.3%) | >0.999 | |
| Female | 2 (28.6%) | 2 (25.0%) | 1 (14.3%) | 5 (22.7%) | ||
| Ethnicity | ||||||
| Hispanic or Latino | 1 (14.3%) | 1 (12.5%) | 1 (14.3%) | 3 (13.6%) | 0.906 | |
| Not Hispanic or Latino | 6 (85.7%) | 7 (87.5%) | 5 (71.4%) | 18 (81.8%) | ||
| Not reported | 0 | 0 | 1 (14.3%) | 1 (4.5%) | ||
| Race | ||||||
| Black or African American | 0 | 0 | 1 (14.3%) | 1 (4.5%) | 0.299 | |
| White or Caucasian | 6 (85.7%) | 7 (87.5%) | 3 (42.9%) | 16 (72.7%) | ||
| Not reported | 1 (14.3%) | 1 (12.5%) | 3 (42.9%) | 5 (22.7%) | ||
| Diagnosis group | ||||||
| PSC | 2 (28.6%) | 3 (37.5%) | 5 (71.4%) | 10 (45.5%) | 0.316 | |
| PSC/AIH overlap | 5 (71.4%) | 5 (62.5%) | 2 (28.6%) | 12 (54.5%) | ||
| IBD diagnosis at study entry | ||||||
| Present | 6 (85.7%) | 7 (87.5%) | 6 (85.7%) | 19 (86.4%) | >0.999 | |
| Absent | 1 (14.3%) | 1 (12.5%) | 1 (14.3%) | 3 (13.6%) | ||
| On immumosuppressive medication | ||||||
| Yes | 3 (42.9%) | 4 (50.0%) | 2 (28.6%) | 9 (40.9%) | 0.862 | |
| No | 4 (57.1%) | 4 (50.0%) | 5 (71.4%) | 13 (59.1%) | ||
| Immunosuppressive medication type | ||||||
| Corticosteroids | 1 (14.3%) | 0 | 1 (14.3%) | 2 (9.1%) | 0.644 | |
| Others | 2 (28.6%) | 4 (50.0%) | 1 (14.3%) | 7 (31.8%) | ||
| N/A | 4 (57.1%) | 4 (50.0%) | 5 (71.4%) | 13 (59.1%) | ||
| Height z score | ||||||
| n | 7 | 7 | 7 | 21 | 0.018 | 0.016 (null vs. flare) |
| Mean (SD) | −0.5 (1.0) | 0.6 (0.4) | −0.2 (0.5) | −0.0 (0.8) | 0.432 (null vs. indeterminant) | |
| 0.008 (flare vs. indeterminant) | ||||||
| Weight z score | ||||||
| n | 7 | 7 | 7 | 21 | 0.301 | |
| Mean (SD) | −0.3 (0.9) | 0.4 (0.5) | 0.0 (1.0) | 0.0 (0.8) | ||
| BMI z score | ||||||
| n | 7 | 7 | 7 | 21 | 0.991 | |
| Mean (SD) | 0.006 (0.6) | 0.022 (0.6) | −0.033 (1.1) | −0.001 (0.7) | ||
| Time since diagnosis (years) | ||||||
| n | 7 | 8 | 7 | 22 | 0.063 | |
| Mean (SD) | 3.0 (1.6) | 1.3 (1.2) | 1.9 (1.2) | 2.0 (1.5) | ||
| UDCA dose per kg | ||||||
| n | 6 | 7 | 7 | 20 | 0.34 | |
| Mean (SD) | 13.0 (2.5) | 14.8 (3.4) | 11.8 (4.6) | 13.2 (3.7) | ||
| UDCA dose ranges (mg/kg/day) | ||||||
| >30 | 1 (14.3%) | 0 | 0 | 1 (4.5%) | 0.383 | |
| 20‐30 | 0 | 1 (12.5%) | 0 | 1 (4.5%) | ||
| 13‐20 | 1 (14.3%) | 4 (50.0%) | 2 (28.6%) | 7 (31.8%) | ||
| <13 | 5 (71.4%) | 3 (37.5%) | 5 (71.4%) | 13 (59.1%) | ||
*
Overall P value computed using ANOVA for continuous data and chi‐square or Fisher exact test for categorical data to test any difference between UDCA subgroups.
†
P value for pairwise comparisons computed using two‐sample t test or Wilcoxon sign rank test for continuous data and chi‐square or Fisher exact test for categorical data between each pair of UDCA subgroups. Pairwise comparisons shown only if overall P value is significant.
Abbreviations: BMI, body mass index; N/A, not applicable.