Division of Gastroenterology & Hepatology, University of Calgary, Calgary, Alberta, Canada; Robarts Clinical Trials, Inc, London, Ontario, Canada.
Robarts Clinical Trials, Inc, London, Ontario, Canada; Division of Gastroenterology, University of California San Diego, La Jolla, California.
Clin Gastroenterol Hepatol. 2020 Jul;18(8):1760-1768.e1. doi: 10.1016/j.cgh.2019.09.021. Epub 2019 Sep 20.
BACKGROUND & AIMS: Little is known about the association between rectal bleeding and increased stool frequency with endoscopic findings in patients with mild to moderate ulcerative colitis (UC). We evaluated the associations between rectal bleeding or stool frequency and endoscopic remission in this population.
We performed a post-hoc analysis of data from a phase 3 non-inferiority trial of 817 adults with mild to moderate UC who received treatment with mesalazine. We obtained information on rectal bleeding, stool frequency, and Mayo endoscopic subscores (MESs) at weeks 0, 8, and 38. The sensitivity, specificity, and positive and negative predictive values with which rectal bleeding and stool frequency identified patients with MESs of 0 and/or 1 were calculated at weeks 8 and 38 of treatment. The associations between change in rectal bleeding and stool frequency and change in MES after treatment were quantified using the Spearman's rank correlation coefficient.
Among patients with a MES of 0, 7/82 patients (9%) had a rectal bleeding score of 1 or more and 40/82 patients (49%) had a stool frequency score of 1 or more at week 8; at week 38, 6/167 patients (4%) had a rectal bleeding score of 1 or more and 63/167 patients (38%) had a stool frequency score of 1 or more. Among patients with MESs of 0 or 1, 50/310 patients (16%) had a rectal bleeding score of 1 or more and 162/310 patients (52%) had had a stool frequency score of 1 or more at week 8; at week 38, 18/363 patients (5%) had a rectal bleeding score of 1 or more and 141/363 patients (39%) had a stool frequency score of 1 or more. The Spearman rank correlation coefficients for change in rectal bleeding and stool frequency with change in MES at week 8 were 0.39 (95% CI, 0.32-0.45) and 0.34 (95% CI, 0.27-0.40), respectively. In patients with reduced MESs at week 8, 39/389 patients (10%) had unchanged or worsening rectal bleeding and 81/389 patients (21%) had unchanged or increasing stool frequencies.
In a post-hoc analysis of data from a phase 3 trial of adults with mild to moderate UC treated with mesalazine, we found absence of rectal bleeding to identify patients in endoscopic remission. However, many patients in remission still have increased stool frequency, indicating that it may not be a sensitive marker of disease activity in patients with mild to moderate UC.
对于轻度至中度溃疡性结肠炎（UC）患者，直肠出血与内镜检查结果中粪便频率增加之间的关系知之甚少。我们评估了直肠出血或粪便频率与该人群内镜缓解之间的关联。
我们对 817 名轻度至中度 UC 成年患者的 3 期非劣效性试验数据进行了事后分析，这些患者接受了美沙拉嗪治疗。我们在第 0、8 和 38 周时获得了直肠出血、粪便频率和 Mayo 内镜亚评分（MESs）的信息。在第 8 和 38 周治疗时，计算了直肠出血和粪便频率与 MESs 为 0 和/或 1 的患者的灵敏度、特异性、阳性和阴性预测值。使用 Spearman 等级相关系数量化治疗后直肠出血和粪便频率变化与 MES 变化之间的关联。
在 MES 为 0 的患者中，82 例患者中有 7 例（9%）在第 8 周时直肠出血评分≥1，82 例患者中有 40 例（49%）在第 8 周时粪便频率评分≥1；在第 38 周时，167 例患者中有 6 例（4%）直肠出血评分≥1，167 例患者中有 63 例（38%）粪便频率评分≥1。在 MESs 为 0 或 1 的患者中，310 例患者中有 50 例（16%）直肠出血评分≥1，310 例患者中有 162 例（52%）粪便频率评分≥1，在第 8 周时；在第 38 周时，363 例患者中有 18 例（5%）直肠出血评分≥1，363 例患者中有 141 例（39%）粪便频率评分≥1。第 8 周时直肠出血和粪便频率变化与 MES 变化的 Spearman 秩相关系数分别为 0.39（95%CI，0.32-0.45）和 0.34（95%CI，0.27-0.40）。在第 8 周 MES 降低的患者中，389 例患者中有 39 例（10%）直肠出血无变化或加重，389 例患者中有 81 例（21%）粪便频率无变化或增加。
在对接受美沙拉嗪治疗的轻度至中度 UC 成年患者的 3 期试验数据进行的事后分析中，我们发现无直肠出血可识别内镜缓解的患者。然而，许多缓解的患者仍有增加的粪便频率，表明它可能不是轻度至中度 UC 患者疾病活动的敏感标志物。
