Article Text
Abstract
Introduction In 2018, our Trust approved the use of Vedolizumab in children with Ulcerative Colitis (UC) and Ustekinumab in children with Crohn’s Disease (CD). At the time, access to these drugs for children was only possible through research studies.
Aim Our aim was to assess the efficacy and safety of these novel treatments in our cohort.
Methods We conducted an observational single centre cohort study. Data was obtained from our electronic system, Cerner Millennium, and Infoflex database. Analysis was performed using SPSS.
Results 27 children were treated with Vedolizumab or Ustekinumab with 1 receiving both. All patients had failed anti-TNF medication, except 1 research patient who commenced on Vedolizumab at diagnosis. All patients underwent endoscopy prior to initiating Vedolizumab or Ustekinumab.
Clinical remission was defined as PUCAI<10 and PCDAI<10. There was a higher induction rate of remission than quoted in adult studies with similar maintenance of remission. At 2 years follow up, 55% (15/27) remained in remission, on treatment. 1/27 is currently still on Ustekinumab with mildly active CD and 1/27 had their Vedolizumab stopped due to compliance and monitoring issues. All 10 children currently receiving Vedolizumab remain in remission. 5/6 currently on Ustekinumab remain in remission. Of the 10/27 who failed treatment, 50% were primary non-responders and 50% had secondary loss of response. 9/10 required subtotal colectomy and ileostomy, while the research patient, who was anti-TNF naïve, switched to Infliximab after failing Vedolizumab.
There were no serious adverse events apart from one patient who developed eosinophilic pneumonitis, but it is unclear whether this was due to Vedolizumab or 5 ASA. Minor skin or upper respiratory tract infections were diagnosed in 5/10 patients on Ustekinumab and 1/10 patient developed Clostridium difficile. Adrenal insufficiency, as a result of prolonged courses of steroids, was detected in 7/27 children.
Conclusion In children with refractory IBD failing anti-TNF treatment, Vedolizumab and Ustekinumab are effective and safe alternatives for inducing and maintaining remission, avoiding major invasive surgery.