Pharmacologic treatment of inflammatory bowel disease: Real world outcomes and optimizing strategies

Inflammatory Bowel Disease (IBD) is a chronic disorder of the gastrointestinal tract, consisting of Crohn’s Disease (CD) and Ulcerative Colitis (UC). The last few decades, the treatment options for IBD have expanded with different mechanisms of actions. To assess the efficacy of newly developed drugs, randomized controlled trials (RCT) are the gold standard. However, it has been shown that only one third of IBD patients meet the in- and exclusion criteria of RCT’. This makes Real World Evidence (RWE) of additional value. In Chapter 2, the strengths, limitations and the impact of RWE in IBD are described. RWE can answer research questions efficiently, can inform on safety or comparative effectiveness and is known for its generalizability. We found that RWE can complement outcomes of RCT’s and is fundamental in supporting the decision-making process to improve the outcomes of IBD patients. Furthermore, we showed that ustekinumab, tofacitinib and vedolizumab subcutaneous are effective and safe treatment options in patients with IBD.
In part II of this thesis we described treatment strategies optimizing outcomes in IBD patients.
In patients with moderate-to-severely active IBD, treatment positioning can be challenging. Using our ICC registry, tofacitinib was associated with superior effectiveness outcomes up to one year of treatment compared with vedolizumab in anti-TNF-experienced patients with UC along with comparable safety outcomes.
Furthermore, we studied whether ustekinumab levels at week eight were predictive for biochemical outcomes and saw that statistically significant more patients with UST levels in the highest quartile (>6.3 µg/mL) achieved biochemical remission at week 24. At last, we assessed patient preferences in their UC treatment. We found that the chance of symptom reduction after one year was the most important attribute in choosing between treatments in UC.