Institute of Paediatric Gastroenterology, Hebrew University of Jerusalem, Jerusalem, Israel.
Soroka University Medical Center and Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.
J Crohns Colitis. 2017 May 1;11(5):527-533. doi: 10.1093/ecco-jcc/jjw180.
Trials in adults suggested that, in ulcerative colitis [UC], once-daily [OD] dosing of 5-ASA [5-amino salicylic acid] may be as or more effective than twice-daily [BD] dosing. In this induction of remission, investigator-blinded, randomised controlled-trial, we aimed to compare effectiveness and safety of once- versus twice-daily mesalazine in paediatric UC.
Children, aged 4-18 years with a PUCAI [Paediatric Ulcerative Colitis Activity Index] of 10-55 points at inclusion, were randomised in blocks of six with blinded allocation to OD or BD mesalazine, using a weight-based dosing table. The primary outcome was mean PUCAI score at Week 6.
A total of 83/86 randomised children were eligible and analysed: 43 in the OD group and 40 in the BD group (mean age 14 ± 2.7 years, 43 [52%] males, 51 [62%] extensive colitis). The groups did not differ with regard to disease activity or any other parameter at baseline. There was no difference in median PUCAI score between the OD group and BD group at Week 6: 15 ( interquartile range [IQR] 5-40) versus 10 [0-40]; p = 0.48]. Response was seen in 25 [60%] OD versus 25 [63%] BD dosing [p = 0.78]. Proportion of children in remission [PUCAI < 10] at Week 6 was 13 [30%] OD versus 16 [40%] BD; p = 0.35]. Most adverse events were related to disease aggravation; the rates of serious adverse events were similar [p > 0.2].
In this first randomised controlled trial in children, no differences were found between OD and BD dosing for any clinical outcome. Remission was achieved in 35% of children treated with mesalazine for active UC.
成人临床试验表明，在溃疡性结肠炎[UC]中，每日一次[OD]剂量的 5-ASA[5-氨基水杨酸]可能与每日两次[BD]剂量一样有效，甚至更有效。在本次缓解诱导研究中，我们旨在比较儿童 UC 中每日一次与每日两次美沙拉嗪的有效性和安全性，采用盲法、随机对照试验。
纳入时 PUCAI[儿童溃疡性结肠炎活动指数]为 10-55 分的 4-18 岁儿童，按区组（每组 6 例）随机分配接受 OD 或 BD 美沙拉嗪治疗，采用基于体重的剂量表。主要结局是第 6 周时平均 PUCAI 评分。
共有 83/86 名随机入组的儿童符合条件并进行了分析：OD 组 43 例，BD 组 40 例（平均年龄 14±2.7 岁，43[52%]为男性，51[62%]为广泛性结肠炎）。两组在基线时的疾病活动度或其他任何参数均无差异。第 6 周时 OD 组和 BD 组的中位数 PUCAI 评分无差异：15（四分位距[IQR]5-40）与 10[0-40]；p=0.48]。OD 组和 BD 组的缓解率分别为 25[60%]和 25[63%]；p=0.78]。第 6 周时达到缓解（PUCAI<10）的儿童比例分别为 13[30%]和 16[40%]；p=0.35]。大多数不良事件与疾病加重有关；严重不良事件发生率相似[p>0.2]。
在这项儿童首次随机对照试验中，OD 与 BD 剂量在任何临床结局方面均无差异。用美沙拉嗪治疗活动性 UC 的儿童中，35%达到缓解。
