(1) Background: Evidence on the outcomes of ustekinumab treatment in real-world Crohn's disease (CD) patients is needed. Our aim was to evaluate the effectiveness and safety of ustekinumab in CD, reported by observational studies. (2) Methods: bibliographical searches were performed (PubMed, EMBASE).
Selection: observational studies assessing the effectiveness and safety of ustekinumab in CD.
Exclusion criteria: studies using ustekinumab as a prophylaxis for postoperative recurrence or perianal disease.
Data synthesis: effectiveness by intention-to-treat (random-effects model). Data were stratified by study design, population included, administered dose, and prior biologic exposure. (3) Results: A total of 63 studies (8529 patients) were included. Response was achieved in 60% (95% CI, 54-67%) in the short term (8-14 weeks); 64% (57-71%) in the medium term (16-24 weeks); and 64% (52-74%) in the long term (48-52 weeks). Remission was achieved in 37% (28-46%) in the short term; 42% (36-49%) in the medium term; and 45% (37-53%) in the long term. The endoscopic remission rate was 33% (25-40%) in the long term. Eighteen percent of patients lost response during follow-up. Nearly one-third of the patients needed dose optimisation, and in 59% of them it was effective. Twenty-five percent of patients developed adverse events, leading to treatment withdrawal in seven percent of the cases. (4) Conclusions: Ustekinumab is an effective and safe therapy in real-world refractory CD patients. Dose optimisation is frequently required, being effective in a high percentage of cases.
Keywords: Crohn’s disease; effectiveness; real-world evidence; safety; ustekinumab.