Table 3.
Baseline and Week 4 clinical and demographic characteristics grouped by Week 12 outcome
| Week 12 CS-Free Remission (n=141) | Neither CS-Free Remission nor Additional Therapy/Colectomy (n=193) | Additional Therapy/Colectomy (n=82) | p-value | |
|---|---|---|---|---|
|
| ||||
| Age (years) (mean±SD) | 12.2 ± 3.5 | 12.9 ± 3.1 | 12.9 ± 3.1 | 0.11 |
|
| ||||
| Female (%) | 72 (51%) | 91 (47%) | 44 (54%) | 0.57 |
|
| ||||
| Nonwhite race (%) | 24/139 (17%) | 26/190 (14%) | 15/79 (19%) | 0.48 |
|
| ||||
| BMI z score (mean±SD) | −0.1 ± 1.2 | −0.2 ± 1.2 | −0.5 ± 1.6 | 0.13 |
|
| ||||
| Hospitalized at baseline (%) | 33 (23%) | 73 (38%) | 55 (67%) | <0.0001 ** |
|
| ||||
| Baseline Characteristics: | ||||
|
| ||||
| PUCAI total score (range 0–85 by 5) (mean±SD) | 42.0 ± 19.3 | 50.6 ± 18.4 | 62.1 ± 17.2 | <0.0001 ** |
| 10–30 (Mild) | 50 (35%) | 42 (22%) | 6 (7%) | <0.0001 ** |
| 35–60 (Moderate) | 62 (44%) | 90 (47%) | 30 (37%) | |
| ≥65 (Severe) | 29 (21%) | 61 (32%) | 46 (56%) | |
|
| ||||
| Abdominal pain | 108 (77%) | 163 (84%) | 73 (89%) | 0.041 * |
| Diarrhea | 121 (86%) | 181 (94%) | 82 (100%) | 0.0004 ** |
| Rectal bleeding | 127 (90%) | 181 (94%) | 78 (95%) | 0.29 |
|
| ||||
| Mayo total score (range 0–12) (mean±SD) | 7.0 ± 2.4 | 7.7 ± 2.3 | 9.3 ± 2.2 | <0.0001 ** |
| % ≥10 | 23 (16%) | 51 (26%) | 42 (51%) | <0.0001 ** |
| % ≥11 | 10 (7%) | 23 (12%) | 36 (44%) | <0.0001 ** |
|
| ||||
| Mayo endoscopy score (range 0–3) (mean±SD) | 2.1 ± 0.7 | 2.1 ± 0.7 | 2.5 ± 0.5 | <0.0001 ** |
|
| ||||
| Partial Mayo score (range 0–9) (mean±SD) | 4.9 ± 2.0 | 5.6 ± 1.9 | 6.8 ± 1.9 | <0.0001 ** |
|
| ||||
| Disease Extent: Proctosigmoiditis | 13 (9%) | 15 (8%) | 0 (0%) | <0.0004 ** |
| Left-sided colitis | 21 (15%) | 19 (10%) | 3 (4%) | |
| Extensive/Pancolitis/Unassessable | 107 (76%) | 159 (82%) | 79 (96%) | |
|
| ||||
| Relative rectal sparing | 19/141 (13%) | 14/192 (7%) | 4/82 (5%) | 0.053 |
|
| ||||
| Cecal patch | 16/135 (12%) | 9/179 (5%) | 4/72 (6%) | 0.059 |
|
| ||||
| Non-specific macroscopic gastritis | 26/137 (19%) | 53/189 (28%) | 31/82 (38%) | 0.0089 * |
|
| ||||
| Rectal biopsy eosinophilic inflammation (count > 32/hpf) | 73/125 (58%) | 105/162 (65%) | 26/69 (38%) | 0.0007 ** |
|
| ||||
| Rectal biopsy surface villiform changes | 35/125 (28%) | 57/161 (35%) | 39/68 (57%) | 0.0002 ** |
|
| ||||
| Rectal biopsy basal plasmacytosis | 52/116 (45%) | 86/152 (57%) | 32/58 (55%) | 0.14 |
|
| ||||
| Hemoglobin (g/dL) | N=131 | N=179 | N=81 | |
| Mean±SD | 11.9 ± 1.9 | 11.5 ± 2.2 | 10.5 ± 2.4 | <0.0001 ** |
| % <10 g/dL | 19 (15%) | 42 (23%) | 35 (43%) | <0.0001 ** |
|
| ||||
| Platelet count (x109/L) | N=130 | N=178 | N=80 | |
| Median (P25, P75) | 362 (286,434) | 355 (297,473) | 410 (345,496) | 0.0092 * |
| >500 | 18 (14%) | 38 (21%) | 20 (25%) | 0.10 |
|
| ||||
| WBC (x109/L) | N=127 | N=178 | N=81 | |
| Median (P25, P75) | 8.9 (7.2,11.3) | 9.1 (7.0,11.4) | 10.6 (8.0,13.8) | 0.0048 * |
| % > 12 (x109/L) | 23 (18%) | 42 (24%) | 31 (38%) | 0.0040 * |
|
| ||||
| ESR (mm/hr) | N=128 | N=171 | N=75 | |
| Median (P25, P75) | 22 (11,39) | 23 (12,38) | 36 (22,53) | 0.0002 ** |
| % ≤20 mm/hr | 61 (48%) | 82 (48%) | 17 (23%) | 0.0002 ** |
| % >40 mm/hr | 30 (23%) | 38 (22%) | 31 (41%) | |
|
| ||||
| CRP or hsCRP > 2x ULN | 24/101 (24%) | 37/142 (26%) | 34/67 (51%) | <0.0001 ** |
|
| ||||
| Albumin (g/dL) | N=140 | N=189 | N=81 | |
| Mean±SD | 3.9 ± 0.6 | 3.6 ± 0.7 | 3.3 ± 0.7 | <0.0001 ** |
| % <3.5 g/dL | 32 (23%) | 65 (34%) | 40 (49%) | 0.0003 ** |
|
| ||||
| Fecal calprotectina (mcg/g) | N=78 | N=110 | N=48 | |
| Median (P25, P75) | 2195 (1202, 3663) | 2002 (1180, 4043) | 3015 (1532, 3892) | 0.43 |
|
| ||||
| Fecal OPG (pg/mL)a | N=54 | N=90 | N=33 | |
| Median (P25, P75) | 119 (31, 1460) | 508 (31, 3220) | 1208 (205, 6427) | 0.015 * |
| % >1000 pg/mL | 14 (26%) | 39 (43%) | 17 (52%) | 0.049 * |
|
| ||||
| 25-OH Vitamin D (ng/mL) | N=133 | N=173 | N=75 | |
| Median (P25, P75) | 28.8 (24.5,35.3) | 28.8 (24.8,35.7) | 26.6 (22.2,31.1) | 0.047 * |
| % < 20 Deficient | 12 (9%) | 16 (9%) | 11 (15%) | 0.37 |
|
| ||||
| ANCA | N=134 | N=174 | N=77 | |
| % Positive titer | 89 (66%) | 110 (63%) | 54 (70%) | 0.56 |
| % Titer ≥100 EU/ml | 22 (16%) | 33 (19%) | 19 (25%) | 0.34 |
|
| ||||
| OmpC (EU/ml) | N=134 | N=174 | N=77 | |
| Median (P25, P75) | 7.1 (4.9,10.4) | 7.0 (5.3,10.3) | 8.8 (6.3,13.2) | 0.014 * |
| % Positive titer | 5 (4%) | 12 (7%) | 3 (4%) | 0.45 # |
| % titer ≥12 EU/ml | 24 (18%) | 33 (19%) | 22 (29%) | 0.14 |
|
| ||||
| Week 4 Characteristics: | ||||
|
| ||||
| Week 4 Remission | 109 (77%) | 91 (47%) | 8 (10%) | <0.0001 ** |
|
| ||||
| Week 4 Remission w/fecal calprotectina <250 mcg/g | 35/100 (35%) | 19/135 (14%) | 1/46 (2%) | <0.0001 ** |
|
| ||||
| Week 4 additional therapy/colectomy | 0 (0%) | 0 (0%) | 45 (55%) | <0.0001 ** |
|
| ||||
| Week 4 PUCAI score (range 0–85 by 5) | N=138 | N=193 | N=76 | |
| Mean ± SD | 4.8 ± 7.3 | 12.6 ± 15.6 | 20.1 ± 20.0 | <0.0001 ** |
|
| ||||
| Week 4 partial Mayo score (range 0–9) | N=120 | N=168 | N=67 | |
| Mean ± SD | 0.7 ± 1.1 | 1.5 ± 1.7 | 2.3 ± 2.2 | <0.0001 ** |
|
| ||||
| Week 4 Albumin (g/dL) | N=104 | N=149 | N=67 | |
| Mean ± SD | 4.3 ± 0.4 | 4.2 ± 0.5 | 3.8 ± 0.6 | < 0.0001 ** |
|
| ||||
| Week 4 CRP or hsCRP > 2x ULN | 1/85 (1%) | 6/109 (6%) | 9/55 (16%) | 0.0018 * # |
|
| ||||
| Week 4 ESR (mm/hr) | N=101 | N=135 | N=66 | |
| Median (P25, P75) | 10 (6,17) | 9 (6,18) | 18 (8,35) | 0.0008 ** |
|
| ||||
| Week 4 Fecal calprotectina (mcg/g) | N=100 | N=135 | N=46 | |
| Median (P25, P75) | 476 (171,1432) | 989 (237, 1754) | 1862 (1151, 3659) | <0.0001 ** |
*
p<0.05,
**
p < 0.001.
P-values comparing groups are from a chi-squared or Fisher’s exact test (noted by #) for categorical variables, a Mantel-Haenszel chi-squared test for ordinal variables, and ANOVA or Kruskal-Wallis test for continuous variables. P25, P75: 25th and 75% percentile
a
Results limited to stool collected prior to 4 days of initial treatment, and before or after colonoscopy cleanout.
Additional parameters are presented in Supplemental Table S7.