Table 1.
Characteristics of PROTECT treated population by initial therapy
| Total (N=428) | 5-ASA (N=136) | Oral CS (N=144) | IV CS (N=148) | p-value | |
|---|---|---|---|---|---|
|
| |||||
| Age (mean±SD) | 12.7±3.3 | 12.8±3.3 | 12.5±3.3 | 12.7±3.2 | 0.73 |
| % ≥12 years | 289 (68%) | 92 (68%) | 96 (67%) | 101 (68%) | |
|
| |||||
| Female (%) | 212 (50%) | 69 (51%) | 61 (42%) | 82 (55%) | 0.079 |
|
| |||||
| Non-white (%) | 69/420 (16%) | 21/134 (16%) | 18/140 (13%) | 30/146 (21%) | 0. 21 |
|
| |||||
| Hispanic/Latino (%) | 38/424 (9%) | 18/134 (13%) | 9/143 (6%) | 11/147 (7%) | 0.085 |
|
| |||||
| Weight z-score (mean±SD) | −0.1±1.2 | 0.0±1.1 | 0.0±1.2 | −0.3±1.2 | 0.037 * |
|
| |||||
| Height z-score (mean±SD) | 0.1±1.0 | 0.1±1.0 | 0.2±0.9 | −0.0±1.0 | 0.052 |
|
| |||||
| BMI z-score (mean±SD) | −0.2 ± 1.3 | −0.0 ± 1.2 | −0.2 ± 1.3 | −0.4 ± 1.4 | 0.035 * |
|
| |||||
| Hospitalized at baseline (%) | 166 (39%) | 4 (3%) | 14 (10%) | 148 (100%) | <0.0001 ** |
|
| |||||
| Disease extent | <0.0001 ** | ||||
| Proctosigmoiditis | 29 (7%) | 22 (16%) | 4 (3%) | 3 (2%) | |
| Left-sided colitis | 44 (10%) | 28 (21%) | 14 (10%) | 2 (1%) | |
| Extensive/Pancolitis/Unassessable a | 355 (83%) | 86 (63%) | 126 (88%) | 143 (97%) | |
|
| |||||
| PUCAI (mean±SD) (range 0–85 by 5) | 50.0±19.9 | 31.1±13.3 | 50.4±13.8 | 66.9±13.7 | <0.0001 ** |
| % 10–30 (Mild) | 102 (24%) | 84 (62%) | 15 (10%) | 3 (2%) | |
| % 35–60 (Moderate) | 185 (43%) | 50 (37%) | 96 (67%) | 39 (26%) | |
| % ≥65 (Severe) | 141 (33%) | 2 (1%) | 33 (23%) | 106 (72%) | |
|
| |||||
| Abdominal pain | 354 (83%) | 97 (71%) | 122 (85%) | 135 (91%) | <0.0001 ** |
| Diarrhea | 393 (92%) | 104 (76%) | 142 (99%) | 147 (99%) | <0.0001 ** |
| Rectal bleeding | 398 (93%) | 117 (86%) | 136 (94%) | 145 (98%) | <0.0001 ** |
|
| |||||
| Total Mayo score (mean±SD) (range 0–12) | 7.8±2.5 | 5.5±1.8 | 7.9±1.7 | 9.8±1.7 | <0.0001 ** |
| % 0–6 | 125 (29%) | 99 (73%) | 23 (16%) | 3 (2%) | |
| % 7–9 | 183 (43%) | 35 (26%) | 93 (65%) | 55 (37%) | |
| % ≥10 | 120 (28%) | 2 (1%) | 28 (19%) | 90 (61%) | |
|
| |||||
| Total Mayo score % ≥11 | 71 (17%) | 0 (0%) | 6 (4%) | 65 (44%) | <0.0001 ** |
|
| |||||
| Mayo endoscopy sub-score (Mean±SD) (range 0–3) | 2.2±0.7 | 1.8±0.6 | 2.2±0.6 | 2.6±0.5 | <0.0001 ** |
| % Mayo 1 | 59 (14%) | 43 (32%) | 13 (9%) | 3 (2%) | |
| % Mayo 2 | 224 (52%) | 79 (58%) | 88 (61%) | 57 (39%) | |
| % Mayo 3 | 145 (34%) | 14 (10%) | 43 (30%) | 88 (59%) | |
|
| |||||
| Relative rectal sparing | 38/427 (9%) | 17/136 (13%) | 15/144 (10%) | 6/147 (4%) | 0.034 * |
|
| |||||
| Macroscopic patchiness | 37/427 (9%) | 23/136 (17%) | 8/144 (6%) | 6/147 (4%) | 0.0002 ** |
|
| |||||
| Cecal patch | 29/397 (7%) | 17/135 (13%) | 10/136 (7%) | 2/126 (2%) | 0.0029 * |
|
| |||||
| Non-specific macroscopic gastritis | 114/420 (27%) | 33/131 (25%) | 28/143 (20%) | 53/146 (36%) | 0.0050 * |
|
| |||||
| Microscopic gastritis | 242/418 (58%) | 59/129 (46%) | 86/142 (61%) | 97/147 (66%) | 0.0023 * |
|
| |||||
| Rectal biopsy eosinophilic inflammation (count > 32/hpf) | 210/367 (57%) | 64/116 (55%) | 82/125 (66%) | 64/126 (51%) | 0.052 |
|
| |||||
| Rectal biopsy surface villiform changes | 135/364 (37%) | 31/115 (27%) | 47/125 (38%) | 57/124 (46%) | 0.010 * |
|
| |||||
| Rectal biopsy basal plasmacytosis | 176/336 (52%) | 51/111 (46%) | 65/118 (55%) | 60/107 (56%) | 0.25 |
|
| |||||
| Hemoglobin g/dL | N=402 | N=122 | N=133 | N=147 | |
| Mean ± SD | 11.4 ± 2.2 | 12.5 ± 1.8 | 11.5 ± 2.1 | 10.5 ± 2.2 | <0.0001 ** |
| % <10 g/dL | 98 (24%) | 8 (7%) | 28 (21%) | 62 (42%) | |
| % <12 g/dL | 231 (58%) | 46 (38%) | 77 (58%) | 108 (73%) | |
|
| |||||
| Platelet count (x109/L) | N=399 | N=121 | N=133 | N=145 | |
| Median (P25, P75) | 372 (303,462) | 319 (251,406) | 376 (318,464) | 411 (339,509) | <0.0001 ** |
| >500 | 77 (19%) | 10 (8%) | 28 (21%) | 39 (27%) | 0.0005 ** |
|
| |||||
| White blood count (x109/L) | N=397 | N=119 | N=133 | N=145 | |
| Median (P25, P75) | 9.2 (7.2, 12.1) | 7.8 (6.5, 9.3) | 9.2 (7.0, 11.1) | 11.3 (8.7, 14.9) | <0.0001 ** |
| % >12 x 109/L | 100 (25%) | 10 (8%) | 23 (17%) | 67 (46%) | |
|
| |||||
| ESR (mm/hr) | N=385 | N=118 | N=125 | N=142 | |
| Median (P25, P75) | 25 (12, 42) | 15 (8, 24) | 25 (12, 39) | 38 (21, 57) | <0.0001 ** |
| % ≤20 mm/hr | 165 (43%) | 79 (67%) | 53 (42%) | 33 (23%) | |
| % >40 mm/hr | 102 (26%) | 8 (7%) | 29 (23%) | 65 (46%) | |
|
| |||||
| CRP/hsCRP | N=315 | N=89 | n=102 | N=124 | |
| % >ULN | 144 (46%) | 18 (20%) | 44 (43%) | 82 (66%) | <0.0001 ** |
| % >2x ULN | 97 (31%) | 10 (11%) | 27 (26%) | 60 (48%) | <0.0001 ** |
|
| |||||
| Albumin (g/dL) | N=422 | N=133 | N=142 | N=147 | |
| Mean ± SD | 3.7 ± 0.7 | 4.0 ± 0.7 | 3.7 ± 0.6 | 3.4 ± 0.7 | < 0.0001 ** |
| % <3.5g/dL | 138 (33%) | 25 (19%) | 37 (26%) | 76 (52%) | |
|
| |||||
| Fecal calprotectin (mcg/g) b | N=239 | N=74 | N=75 | N=90 | |
| Median (P25, P75) | 2352 (1202, 3928) | 1692 (851, 3631) | 2663 (1202, 3664) | 3202 (1495, 4384) | 0.030 * |
| % ≥250 mcg/g | 226 (95%) | 64 (86%) | 73 (97%) | 89 (99%) | |
|
| |||||
| Fecal OPG (pg/mL) b | N=178 | N=53 | N=59 | N=66 | |
| Median (P25, P75) | 424 (31, 3259) | 119 (31, 1677) | 318 (31, 1638) | 1491 (169, 6830) | 0.0012 * |
| % >1000 pg/mL | 70 (39%) | 15 (28%) | 18 (31%) | 37 (56%) | |
|
| |||||
| 25-OH Vitamin D (ng/mL) | N=393 | N=125 | N=132 | N=136 | |
| Median (P25, P75) | 28.5 (23.9, 34.8) | 28.1 (23.8, 34.0) | 29.9 (25.2, 36.4) | 27.4 (22.4, 33.0) | 0.032* |
| % <20 ng/mL | 42 (11%) | 14 (11%) | 10 (8%) | 18 (13%) | |
|
| |||||
| ANCA | N=397 | N=125 | N=135 | N=137 | |
| % Positive titer | 259 (65%) | 73 (58%) | 90 (67%) | 96 (70%) | 0.13 |
| % Titer ≥100 EU/ml | 75 (19%) | 17 (14%) | 28 (21%) | 30 (22%) | 0.18 |
|
| |||||
| OmpC | N=397 | N=125 | N=135 | N=137 | |
| Median (P25, P75) | 7.4 (5.3,11.2) | 7.2 (5.1,11.2) | 7.3 (5.7,10.2) | 7.5 (5.3,11.6) | 0.76 |
| % Positive titer | 21 (5%) | 8 (6%) | 7 (5%) | 6 (4%) | 0.76 |
| % ≥12 | 81 (20%) | 25 (20%) | 25 (19%) | 31 (23%) | 0.70 |
|
| |||||
| CBir1 | N=397 | N=125 | N=135 | N=137 | |
| % Positive titer | 76 (19%) | 27 (22%) | 27 (20%) | 22 (16%) | 0.50 |
*
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.
a
Unassessable: severe/fulminant disease at presentation and the clinician performed a flexible sigmoidoscopy for safety concerns.
b
Results limited to stool collected prior to 4 days of initial treatment, and before or after colonoscopy cleanout