Abstract
Background: Vedolizumab (vedo) is effective for induction and maintenance of remission in adults with inflammatory bowel disease (IBD). Pediatric data are still limited, especially for the youngest children with very early onset disease (VEO-IBD). The aim of this study was to assess the safety and efficacy of vedo in VEO-IBD. Methods: We performed a retrospective review of pediatric IBD patients with VEO-IBD (defined as aged <6 years) receiving vedo. Data on demographics, disease behavior, activity, and previous treatments/surgeries were collected. Disease activity was assessed using the pediatric Crohn’s disease (CD) activity index (PCDAI) for CD or pediatric ulcerative colitis (UC) activity index (PUCAI) for UC. Primary outcome was clinical response after induction therapy with vedolizumab (4th dose week). It was defined as a decrease in PCDAI of at least 12.5 points between baseline and 4th dose week for CD, and a decrease in PUCAI of at least 20 points between baseline and this time for UC. Descriptive statistics were performed to analyze the data. Results: The study included 16 patients with VEO-IBD who have received vedo: 4/16 (25%) with CD, and 12/16 (75%) with UC at the median age of diagnosis 33.7 months (6.6 months–4.5 years). Median age at vedo initiation was 6.5 years (2.2–16.5 years). Among the analyzed individuals, 56.25% had failed more than one anti-tumor necrosis factor (TNF) alfa agent. Clinical response at 4th dose week was observed in 9/16 (56.3%) patients: mean baseline PCDAI score was 34.4 ± 1.9 and 10.6 ± 1.8 after induction therapy with vedo, while PUCAI score was 26 ± 6 vs. 18 ± 8, respectively. There was improvement in patients’ nutritional state: at baseline 2/16 (12.5%) children had body mass index (BMI) below 1 percentile and no child had such BMI after induction therapy with vedo. No infusion reactions or serious adverse events/infections were reported. Conclusion: Vedolizumab is safe and effective in the medical management of pediatric patients with VEO-IBD.
Highlights
The incidence of pediatric inflammatory bowel disease (IBD) is increasing worldwide, and the age of onset has become younger [1]
Some of Very early onset IBD (VEO-IBD) patients present with immunodeficiency and have genetic causes of their disease which means that loss of function genetic mutations involving immune and/or cytokines pathways lead to development of intestinal inflammation [3]
Monoclonal antibodies against tumor necrosis factor alpha (TNFα), such as infliximab (IFX) or adalimumab (ADA), are safe and effective in induction and maintenance of remission in moderate to severe pediatric Crohn’s disease (CD) and ulcerative colitis (UC)
Summary
The incidence of pediatric inflammatory bowel disease (IBD) is increasing worldwide, and the age of onset has become younger [1]. Some of VEO-IBD patients present with immunodeficiency and have genetic causes of their disease which means that loss of function genetic mutations involving immune and/or cytokines pathways lead to development of intestinal inflammation [3]. Most of these young patients present with a highly severe course of IBD and require more aggressive treatment due to the failure to both conventional and biological therapies [4]. Results: The study included 16 patients with VEO-IBD who have received vedo: 4/16 (25%) with CD, and 12/16
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