极早期发病的小儿炎症性肠病与晚期发病的小儿 IBD 的疗效比较:儿科IBD转诊中心15年的单中心经验

Francesco Baccelli, Erika Cantarelli, Lucia Del Vecchio, F. Labriola, F. Sbravati, Giulia Bardasi, Roberto Rondelli, P. Alvisi
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Patients with monogenic-IBD or IBD-like disease were excluded. We chose a minimum follow up period of 18 months since the diagnosis or any period in patients with Ulcerative Colitis (UC) who underwent surgery. The two cohorts were homogeneous for extension of disease and clinical severity at diagnosis and were compared for different clinical outcomes. Primary outcomes were the cumulative incidence of biological therapy use and the cumulative incidence of major surgery, as severity disease’ indices. Secondary outcomes were the cumulative incidence of clinical remission, steroid-dependence and the relapse free survival at the last follow up. Results: 29 VEO-IBD cases were compared with 52 ped-IBDs. Median follow up time was 4,9 years. UC were most represented in both groups (83% of VEO-IBD, 81% of ped-IBD). At the end of follow up, the surgical rate was significantly higher for VEO-UC group compared with ped-UC (p 0.018). 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引用次数: 0

摘要

背景:近年来,人们对极早发性(VEO)炎症性肠病(IBD)的研究兴趣日益浓厚,该病的诊断年龄小于 6 岁。之前的研究报告显示,与晚发性小儿 IBD 相比,超早期发病的 IBD 病程更严重,这主要归因于遗传在这一年龄组中起着更大的作用。但也有一些研究对这些结果提出了质疑,并报告了相似的结果。我们的研究旨在验证年龄作为病程严重性预测因素的有效作用。研究方法在这项转诊 IBD 单中心回顾性观察研究中,VEO-IBD 与 6-17 岁诊断的 IBD(儿童 IBD)进行了比较。单基因 IBD 或 IBD 类疾病患者被排除在外。我们选择了至少 18 个月的随访期,或接受过手术的溃疡性结肠炎(UC)患者的任何随访期。两组患者的疾病范围和诊断时的临床严重程度相同,并就不同的临床结果进行了比较。主要结果是使用生物疗法的累积发生率和大手术的累积发生率,作为疾病严重程度指数。次要结果是临床缓解的累积发生率、类固醇依赖性和最后一次随访时的无复发存活率。结果:29 例 VEO-IBD 与 52 例儿童 IBD 进行了比较。中位随访时间为 4.9 年。两组患者中UC占多数(VEO-IBD为83%,ped-IBD为81%)。随访结束时,VEO-UC组的手术率明显高于ped-UC组(P 0.018)。在使用生物疗法方面没有发现差异(45.6% 的 VEO-IBD 与 43.5% 的 ped-IBD;P 0.72)。随访结束时,无复发生存期、类固醇依赖、使用免疫抑制疗法和临床缓解的累积发生率相当。讨论我们的数据质疑了VEO-UC与小儿UC相比预后更差的证据。值得注意的是,我们的研究表明,VEO-UC的手术需求更高。
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Outcome of Very Early Onset Pediatric Inflammatory Bowel Diseases Compared to Later-Onset Pediatric IBD: The 15-Year Single-Center Experience of a Referral Pediatric IBD Unit
Background: In last years, research interest increased for very early onset (VEO) inflammatory bowel disease (IBD), defined by age at diagnosis < 6 years. Previous works reported more severe disease course of VEO-IBD, compared with later-onset pediatric IBD, mainly attributed to a greater role of genetics in this age group. Otherwise, some studies questioned these results and reported comparable outcomes. Our study aimed to verify the effective role of age as a predictive factor of severity in the disease course. Methods: In this referral IBD single-center retrospective observational study , VEO-IBD was compared with IBD diagnosed between 6 and 17 years of age (ped-IBD). Patients with monogenic-IBD or IBD-like disease were excluded. We chose a minimum follow up period of 18 months since the diagnosis or any period in patients with Ulcerative Colitis (UC) who underwent surgery. The two cohorts were homogeneous for extension of disease and clinical severity at diagnosis and were compared for different clinical outcomes. Primary outcomes were the cumulative incidence of biological therapy use and the cumulative incidence of major surgery, as severity disease’ indices. Secondary outcomes were the cumulative incidence of clinical remission, steroid-dependence and the relapse free survival at the last follow up. Results: 29 VEO-IBD cases were compared with 52 ped-IBDs. Median follow up time was 4,9 years. UC were most represented in both groups (83% of VEO-IBD, 81% of ped-IBD). At the end of follow up, the surgical rate was significantly higher for VEO-UC group compared with ped-UC (p 0.018). No difference in biologic therapy use was detected (45,6% VEO-IBD vs 43,5% ped-IBD; p 0.72). Cumulative incidences of relapse free survival, steroid-dependence, use of immunosuppressive therapy and clinical remission at the end of follow up were comparable. Discussion: Our data question the evidence of a worse outcome of VEO-UC, in comparison with ped-UC. Notably, we demonstrate a higher need for surgery in VEO-UC.
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