Real-world Comparative Effectiveness of Ustekinumab vs Anti-TNF in Crohn's Disease With Propensity Score Adjustment: Induction Phase Results From the Prospective, Observational RUN-CD Study.

ABSTRACT

BACKGROUND: In addition to randomized controlled trials (RCTs), real-world studies on the effectiveness of ustekinumab (UST) in Crohn's disease (CD) are required inasmuch as RCTs are usually confined to selected patients, which may not represent everyday clinical practice. Within the framework of the prospective real-world RUN-CD registry, a total of approximately 900 CD patients from 44 inflammatory bowel disease centers from all over Germany starting a new therapy with UST or other biologics were screened for a real-world evidence (RWE) comparison of CD patients with UST vs antitumor necrosis factor (TNF). METHODS: A total of 618 CD patients with a nonrandomized biological therapy were qualified for this induction phase effectiveness RUN-CD study of UST vs anti-TNF. To reduce selection bias in estimations of treatment effects, the propensity score with inverse probability of treatment weighting was implemented. The results were reported as odds ratio (OR) and 95% confidence interval (CI). RESULTS: A total of 339 UST and 279 anti-TNF patients were analyzed. The effectiveness of UST vs anti-TNF in terms of clinical remission (UST 65.4% vs anti-TNF 63.0%; OR, 1.11; 95% CI, 0.71-1.74) and steroid-free remission (UST 51.0% vs anti-TNF 53.8%; OR, 0.94; 95% CI, 0.60-1.47) was comparable at the end of induction therapy. Similar results were observed in the bio-naïve and bio-experienced UST vs anti-TNF groups. For both, the remission rates were higher in the bio-naïve than in the bio-experienced groups (P < .05). CONCLUSIONS: In this prospective, observational RUN-CD study, the RWE head-to-head comparison of UST vs anti-TNF showed similar induction effectiveness in both groups, remarkably higher than those found in prior RCTs.

The higher effectiveness outcome rates observed in patients treated with UST compared with pivotal studies in combination with its known favorable safety profile and an improved HRQoL support UST use as a first-line, advanced therapy in CD.