Shaare Zedek Medical Center, Jerusalem, Israel.
Gastroenterology. 2010 Jun;138(7):2282-91. doi: 10.1053/j.gastro.2010.02.047. Epub 2010 Feb 26.
BACKGROUND & AIMS: In a prospective study of children with severe ulcerative colitis (UC), we aimed to assess outcomes and to identify predictors of nonresponse to intravenous corticosteroids.
A total of 128 children (47% males; 12.9 +/- 3.9 y) hospitalized for severe UC were enrolled from 10 pediatric centers. Clinical and laboratory data and the Pediatric UC Activity Index (PUCAI) were recorded throughout the admission. Patients were followed up for 1 year postdischarge.
Thirty-seven (29%; 95% confidence interval [CI], 22%-37%) children failed intravenous corticosteroids and received, within 10.5 +/- 6.4 days, cyclosporine (n = 1; 3%), colectomy (n = 3; 8%), or infliximab (n = 33; 89%). Several predictors were associated with intravenous corticosteroids failure, but the best model included number of stools, amount of blood, age, and new-onset disease (odds ratio [OR], 1.9; 95% CI, 1.1-3.5; OR, 2.5; 95% CI, 1.3-4.6; OR, 1.2; 95% CI, 1.04-1.36; and OR, 0.27; 95% CI, 0.1-0.7, respectively). The PUCAI, followed closely by the Travis rule, strongly predicted response when compared with other measures (Seo and Lindgren indices, C-reactive protein level, and fecal calprotectin level) (P < .001). Aiming for sensitivity on day 3, a PUCAI greater than 45 screened for patients likely to fail intravenous corticosteroids (negative predictive value, 94%; positive predictive value, 43%; P < .001). Aiming for specificity on day 5, a PUCAI score greater than 70 optimally guided implementation of salvage therapy (positive predictive value, 100%; negative predictive value, 79%; P < .001). Twenty-five of 33 children treated with infliximab responded. The overall cumulative colectomy rate was 9% and 19% by discharge and 1-year, respectively. The day 3 PUCAI score predicted response up to 1 year postdischarge (P < .001; time to salvage therapy).
The PUCAI, calculated on days 3 and 5 of steroid therapy, can identify patients requiring salvage therapy. Infliximab is an effective therapy in steroid-refractory pediatric UC.
在一项对患有严重溃疡性结肠炎（UC）儿童的前瞻性研究中，我们旨在评估结局，并确定对静脉用皮质类固醇无反应的预测因素。
从 10 个儿科中心共纳入 128 名因严重 UC 住院的儿童（47%为男性；12.9±3.9 岁）。在整个住院期间记录临床和实验室数据以及儿科 UC 活动指数（PUCAI）。患者在出院后随访 1 年。
37 名（29%；95%置信区间[CI]，22%-37%）儿童静脉用皮质类固醇治疗失败，并在 10.5±6.4 天内接受环孢素（n=1；3%）、结肠切除术（n=3；8%）或英夫利昔单抗（n=33；89%）治疗。一些预测因素与静脉用皮质类固醇治疗失败相关，但最佳模型包括粪便次数、粪便量、年龄和新发疾病（比值比[OR]，1.9；95%CI，1.1-3.5；OR，2.5；95%CI，1.3-4.6；OR，1.2；95%CI，1.04-1.36；OR，0.27；95%CI，0.1-0.7）。PUCAI 紧随 Travis 规则之后，与其他指标（Seo 和 Lindgren 指数、C 反应蛋白水平和粪便钙卫蛋白水平）相比，强烈预测了反应（P<.001）。在第 3 天以灵敏度为目标时，PUCAI 大于 45 预测患者可能对静脉用皮质类固醇无反应（阴性预测值，94%；阳性预测值，43%；P<.001）。在第 5 天以特异性为目标时，PUCAI 评分大于 70 可最佳指导挽救治疗（阳性预测值，100%；阴性预测值，79%；P<.001）。33 名接受英夫利昔单抗治疗的儿童中有 25 名有反应。总体累计结肠切除术率分别为出院时的 9%和 1 年时的 19%。第 3 天的 PUCAI 评分可预测出院后 1 年的反应（P<.001；挽救治疗时间）。
在皮质类固醇治疗的第 3 天和第 5 天计算的 PUCAI 可识别需要挽救治疗的患者。英夫利昔单抗是治疗皮质类固醇难治性小儿 UC 的有效疗法。
