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.
Solitary rectal ulcer syndrome (SRUS) is a benign rectal disease that is rare in pediatric populations due to its underdiagnosis and misdiagnosis in children. It is often misdiagnosed as malignancy, Crohn's disease, and ulcerative colitis due to its wide and varying clinical presentations. Both SRUS and inflammatory bowel disease (IBD) can present with rectal bleeding, constipation, diarrhea, and abdominal pain. Furthermore, macroscopic ulcers and inflammation can be seen in both diseases, making it difficult to diagnose without a biopsy. We present two cases in the pediatric population whose diagnoses of SRUS were delayed because the symptoms and macroscopic findings initially supported the differential diagnosis of IBD. These cases emphasize the difficulty and importance of differentiating between IBD and SRUS, and should encourage practitioners to include this differential diagnosis earlier on to improve diagnostic accuracy and begin implementing effective treatment. This can eventually decrease overall treatment time, unnecessary surgeries and diagnostic testing, and increase the emotional reassurance of the benign nature of the disease.
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