Vedolizumab Experience in Children and Adolescents With Inflammatory Bowel Disease: A Multicenter Observational Study

Abstract

Background Vedolizumab is increasingly used off-label to treat children and adolescents with inflammatory bowel disease (IBD). In the absence of rigorous clinical trial experience, multicenter observational data are important to establish expectations for efficacy and safety. We examined one-year outcomes following vedolizumab therapy in a large multicenter paediatric IBD cohort. Methods We performed a retrospective study of 159 paediatric patients (4-17 years old) with IBD (78, Crohn disease [CD]; 81, ulcerative colitis/IBD-unspecified [UC/IBD-U]) treated with vedolizumab for 1-year at 8 paediatric medical centers in the United States. Demographics, clinical outcomes, laboratory data, and vedolizumab dosing were recorded. The primary outcome was corticosteroid free clinical remission at 1-year. Other measured outcomes were clinical remission at 12-weeks and/or 24 weeks, laboratory outcomes at 1 year, and endoscopy/histology results at 1 year. Results Among the 159 patients (mean age, 14.5±2.4 y; 86% anti-TNF experienced), 68/159 (43%) achieved corticosteroid free clinical remission at 1 year (CD 35/78, 45%; UC/IBD-U 33/81, 40%). Vedolizumab therapy failed and was discontinued in 33/159 (21%) patients prior to 1-year (CD, 18/78, 23%; UC/IBD-U, 15/81, 19%). While Week 12 clinical remission was not predictive of 1-year clinical remission in either CD or UC/IBD-U, week 24 clinical remission was predictive of 1-year clinical remission only in CD patients. No infusion reactions or serious side effects were noted. Conclusion Vedolizumab was safe and effective in this paediatric population with approximately 43% achieving corticosteroid free clinical remission at 1-year. Similar efficacy was noted in both CD and UC.

Efficacy and safety of vedolizumab for pediatrics with inflammatory bowel disease: a systematic review

“…Of these, thirty-eight were excluded for various reasons (Fig. 1 ), leaving 10 articles including a total of 455 patients ( n = 216 CD, n = 239 UC/IBD-U) [ 22 – 31 ]. All studies focused on both CD and UC/IBD-U.…”

“…All studies focused on both CD and UC/IBD-U. Seven studies reported clinical remission rates [ 22 – 25 , 29 – 31 ]. Four studies reported clinical response rates [ 23 , 25 , 29 , 30 ].…”

“…Four studies reported clinical response rates [ 23 , 25 , 29 , 30 ]. Six studies reported CS-free remission rates [ 22 – 25 , 28 , 31 ] and 3 reported mucosal healing [ 24 , 27 , 29 ]. Nine studies reported safety outcomes for CD or UC/IBD-U combined, rather than by separate indication [ 22 – 26 , 28 – 31 ].…”

“…Six studies reported CS-free remission rates [ 22 – 25 , 28 , 31 ] and 3 reported mucosal healing [ 24 , 27 , 29 ]. Nine studies reported safety outcomes for CD or UC/IBD-U combined, rather than by separate indication [ 22 – 26 , 28 – 31 ]. Characteristics of the included studies are listed in Table 1 , and patient demographics were showed in Table 2 .…”

“…In CD patients, the short-term clinical remission rate at 2 weeks of therapy ranged from 0 to 27% [two studies] [ 22 , 25 ], and at 6 weeks of treatment ranged from 0 to 35% [four studies] [ 22 – 25 ]. For maintenance therapy, the remission rates were ranging from 17 to 42% at 14 weeks [six studies] [ 22 – 25 , 29 , 30 ], 24% to 39% at 22 weeks [three studies] [ 22 – 24 ], 44% at 24 weeks [one study] [ 31 ], 31% to73% at 30 weeks [two studies] [ 22 , 30 ], and 25% to 49% at 1 year [three studies] [ 24 , 30 , 31 ]. In UC/IBD-U patients, the short-term clinical remission rate at 2 weeks of therapy ranged from 40 to 41% [two studies] [ 22 , 25 ], and at 6 weeks of treatment ranged from 20 to 64% [four studies] [ 22 – 25 ].…”