Institute for Maternal and Child Health, IRCCS Burlo Garofolo, Trieste, Italy.
Bureau de biostatistique et d'épidemiologie, Gustave Roussy and Université Paris-Saclay, Paris, France.
Inflamm Bowel Dis. 2024 Jan 5;30(1):20-28. doi: 10.1093/ibd/izad018.
Few drugs have been studied for patients with very early onset inflammatory bowel disease (VEOIBD). This study aimed to evaluate the efficacy and tolerance of thalidomide in children with VEOIBD compared with children with pediatric-onset IBD (pIBD).
A retrospective cohort study with a control group was conducted. Propensity score 1:1 matching was used to identify control subjects. The treatment persistence; the causes of drug withdrawal; the rate of clinical remission and mucosal healing at 1, 2, and 3 years; and adverse events (AEs) were evaluated in children with VEOIBD treated with thalidomide and compared with children with pIBD.
Thirty-nine courses of treatment with thalidomide in VEOIBD and pIBD patients were compared. The treatment persistence at 1, 2, and 3 years was 68.2% (95% confidence interval [CI], 50.8%-80.6%), 57.0% (95% CI, 39.6%-71.1%), and 50.9% (95% CI, 33.7%-65.8%) for VEOIBD patients and 81.7% (95% CI, 65.3%-90.9%), 60.0% (95% CI, 41.7%-74.3%) and 33.0% (95% CI, 17.4%-49.5%) for pIBD patients, respectively (P = .12). A significantly higher proportion of VEOIBD patients discontinued therapy due to lack of efficacy (48.2% vs 17.2%; P = .03), while AEs were the main reason for discontinuation in pIBD patients. Clinical remission and mucosal healing rates did not significantly differ between VEOIBD and pIBD patients. A significatively lower number of VEOIBD patients experienced AEs compared with pIBD patients (14 [35.9%] vs 30 [76.9%]; P = .0005).
Thalidomide is an effective and tolerated treatment in children with VEOIBD. Discontinuation due to lack of efficacy is more frequent, but AEs are less common than in children with pIBD.
很少有药物针对发病非常早的炎症性肠病（VEOIBD）患者进行研究。本研究旨在评估与儿科发病的炎症性肠病（pIBD）患儿相比，沙利度胺治疗 VEOIBD 患儿的疗效和耐受性。
进行了一项回顾性队列研究，并设置了对照组。采用 1:1 倾向评分匹配来识别对照受试者。评估了 VEOIBD 患儿接受沙利度胺治疗后的治疗持续时间；停药原因；1、2 和 3 年时的临床缓解率和黏膜愈合率；以及不良事件（AE），并与 pIBD 患儿进行了比较。
比较了 VEOIBD 和 pIBD 患儿 39 例沙利度胺疗程的治疗情况。VEOIBD 患儿治疗 1、2 和 3 年的治疗持续率分别为 68.2%（95%可信区间[CI]，50.8%-80.6%）、57.0%（95%CI，39.6%-71.1%）和 50.9%（95%CI，33.7%-65.8%），pIBD 患儿的治疗持续率分别为 81.7%（95%CI，65.3%-90.9%）、60.0%（95%CI，41.7%-74.3%）和 33.0%（95%CI，17.4%-49.5%）（P=.12）。VEOIBD 患儿因疗效不佳而停药的比例显著更高（48.2% vs 17.2%；P=.03），而 pIBD 患儿停药的主要原因是 AE。VEOIBD 和 pIBD 患儿的临床缓解率和黏膜愈合率无显著差异。与 pIBD 患儿相比，VEOIBD 患儿发生 AE 的数量显著更少（14 例[35.9%] vs 30 例[76.9%]；P=.0005）。
沙利度胺是 VEOIBD 患儿有效且耐受良好的治疗药物。因疗效不佳而停药的情况更常见，但 AE 比 pIBD 患儿更少。
