Strisciuglio Caterina, Coppola Vincenzo, Russo Marina, Tolone Carlo, Marseglia Gian Luigi, Verrotti Alberto, Caimmi Silvia, Caloisi Claudia, D'Argenio Valeria, Sacchetti Lucia, Staiano Annamaria
Department of Woman, Child and General and Specialized Surgery, University of Campania "Luigi Vanvitelli", Naples, Italy.
Department of Translational Medical Sciences, Section of Pediatric, University of Naples Federico II, Naples, Italy.
Front Pediatr. 2021 Oct 29;9:753938. doi: 10.3389/fped.2021.753938. eCollection 2021.
Polyethylene glycol (PEG) is recommended as first-line treatment of pediatric functional constipation. However, the oral route of administration is often poorly feasible in children mostly due to poor palatability. Promelaxin microenemas exert a topical evacuative action and may offer a valuable option in pediatric FC. To assess whether Promelaxin microenemas would be non-inferior to PEG 4000 in young children with FC. This is a randomized, open-label, multi-centric, non-inferiority trial enrolling infants and young children aged 6-48 months, with FC according to Rome III criteria. After 1 week of run in, children were randomized to 2 weeks of Promelaxin or PEG, followed by a 6-week on-demand treatment period. Primary endpoint was response rate to randomized treatment, with "response" defined as at least 3 evacuations per week and an average increase of at least one evacuation per week as compared to baseline. Safety, stool consistency and the analysis of fecal microbiota were secondary endpoints. Out of the 158 patients who entered the trial, 153 patients were treated (77 and 76, PEG and Promelaxin arm, respectively). In the primary analysis, the 95% confidence interval (CI) for the treatment's effect lay entirely above the non-inferiority margin in both Full Set (FAS) and Per Protocol (PP) analyses, providing evidence of the non-inferiority of Promelaxin vs. PEG 4000 [response rate difference: 16.5% (CI 1.55-31.49%) and 11.03% (CI -5.58 to 27.64%), FAS and PP analyses, respectively]. Mean compliance to the randomized treatment was >80% in both arms. Secondary endpoints did not significantly differ between the two arms, except for the average number of total days of on-demand treatment that was significantly lower in the Promelaxin arm [14.6 (12.7) vs. 9.8 (9.1), mean (SD); primary endpoint responders in PEG and Promelaxin arm, respectively; = 0.027]. Microbiota evenness significantly increased in the PEG 4000 arm at V4 as compared to the Promelaxin arm ( < 0.05). In addition, at V5, patients treated with PEG showed a significantly decreased microbiota density as compared to patients treated with Promelaxin ( = 0.036). Promelaxin microenemas are non-inferior to oral PEG in children with FC. www.ClinicalTrials.gov, identifier: NCT02751411.
聚乙二醇（PEG）被推荐作为小儿功能性便秘的一线治疗药物。然而，口服给药途径在儿童中往往可行性较差，主要原因是口感不佳。普洛美拉辛微灌肠剂具有局部促排便作用，可能为小儿功能性便秘提供一种有价值的选择。为评估普洛美拉辛微灌肠剂在患有功能性便秘的幼儿中是否不劣于聚乙二醇4000。这是一项随机、开放标签、多中心、非劣效性试验，纳入6至48个月、符合罗马III标准的功能性便秘婴幼儿。经过1周的导入期后，将儿童随机分为接受2周普洛美拉辛或聚乙二醇治疗，随后是6周的按需治疗期。主要终点是随机治疗的反应率，“反应”定义为每周至少3次排便，且与基线相比每周平均排便次数至少增加1次。安全性、粪便稠度和粪便微生物群分析为次要终点。在158名进入试验的患者中，153名患者接受了治疗（分别为77名和76名，聚乙二醇组和普洛美拉辛组）。在主要分析中，在全分析集（FAS）和符合方案集（PP）分析中，治疗效果的95%置信区间（CI）完全高于非劣效界值，提供了普洛美拉辛相对于聚乙二醇4000非劣效的证据[反应率差异：分别为16.5%（CI 1.55 - 31.49%）和11.03%（CI - 5.58至27.64%），FAS和PP分析]。两组对随机治疗的平均依从性均>80%。除按需治疗的总天数平均值在普洛美拉辛组显著更低外[分别为14.6（12.7）天和9.8（9.1）天，平均值（标准差）；聚乙二醇组和普洛美拉辛组的主要终点反应者]，两组次要终点无显著差异（P = 0.027）。与普洛美拉辛组相比，聚乙二醇4000组在V4时微生物群均匀度显著增加（P < 0.05）。此外，在V5时，与接受普洛美拉辛治疗的患者相比，接受聚乙二醇治疗的患者微生物群密度显著降低（P = 0.036）。普洛美拉辛微灌肠剂在患有功能性便秘的儿童中不劣于口服聚乙二醇。ClinicalTrials.gov网站，标识符：NCT02751411。
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