Patel Halee, Karam Lina, Kellermayer Richard
Department of Pediatrics, Section of Gastroenterology, Hepatology and Nutrition, Baylor College of Medicine/Texas Children's Hospital, Houston, TX.
Children's Nutrition and Research Center, Houston, TX.
JPGN Rep. 2022 Dec 27;4(1):e276. doi: 10.1097/PG9.0000000000000276. eCollection 2023 Feb.
Vedolizumab is an anti-α4β7 integrin antibody that has been used successfully in the treatment of adult-onset inflammatory bowel diseases (IBDs: Crohn disease [CD] and ulcerative colitis [UC]). Its off-label use in the pediatric IBD (PIBD) population is increasing, but knowledge on durability beyond 6 months of treatment is limited.
A real-life, single-center, retrospective study of PIBD patients treated with vedolizumab was performed. Data on demographics, prior and concomitant treatments, and disease activity were obtained at 14 weeks, 26 weeks, 1 year, and 2 years of therapy. Primary outcome was corticosteroid- and other biologic-free remission (based on pediatric ulcerative colitis activity index [PUCAI]).
Thirty-nine patients were studied. By 1 year, 65% of CD and 68% of UC patients continued on vedolizumab therapy. Corticosteroid- and other biologic-free remission was 29% in CD and 16% in UC. By 2 years, 36% of CD and 47% of UC patients continued therapy. Corticosteroid- and other biologic-free remission was 21% in CD and 40% in UC. By 2 years, 80% of CD and 100% of UC patients were on intensified treatment regimen compared to the manufacturer guidance. Nine patients (23%) required surgical intervention within 26 months of starting vedolizumab indicating the severity of IBD in this cohort.
Vedolizumab is a useful therapeutic modality in PIBD patients refractory to anti-TNF therapy, although with declining effectiveness by 2 years. Intensified treatment regimens are associated with long-term durability. Larger prospective trials in children are warranted.
维多珠单抗是一种抗α4β7整合素抗体，已成功用于治疗成人起病的炎症性肠病（IBD：克罗恩病[CD]和溃疡性结肠炎[UC]）。其在儿科IBD（PIBD）人群中的非标签使用正在增加，但关于治疗6个月以上的持久性的知识有限。
对接受维多珠单抗治疗的PIBD患者进行了一项真实世界、单中心、回顾性研究。在治疗的14周、26周、1年和2年时获取了人口统计学数据、既往和同时进行的治疗以及疾病活动情况。主要结局是无皮质类固醇和其他生物制剂缓解（基于儿科溃疡性结肠炎活动指数[PUCAI]）。
研究了39例患者。到1年时，65%的CD患者和68%的UC患者继续接受维多珠单抗治疗。CD患者的无皮质类固醇和其他生物制剂缓解率为29%，UC患者为16%。到2年时，36%的CD患者和47%的UC患者继续治疗。CD患者的无皮质类固醇和其他生物制剂缓解率为21%，UC患者为40%。到2年时，与制造商指南相比，80%的CD患者和100%的UC患者采用了强化治疗方案。9例患者（23%）在开始维多珠单抗治疗后的26个月内需要手术干预，表明该队列中IBD的严重程度。
维多珠单抗是对抗TNF治疗难治的PIBD患者的一种有用治疗方式，尽管2年后疗效会下降。强化治疗方案与长期持久性相关。有必要在儿童中进行更大规模的前瞻性试验。
