Objective  To evaluate the efficacy, safety and cost-effectiveness of tofacitinib in the treatment of ulcerative colitis using a rapid health technology assessment, and to provide an evidence-based basis for rational clinical use and decision-making.

Methods  PubMed, Embase, Cochrane Library, CNKI, WanFang Data, VIP and relevant HTA websites and databases were electronically searched to collect high-quality clinical evidence and economic evaluation literature of tofacitinib in the treatment of ulcerative colitis from inception to July 31, 2024. The results were qualitatively analyzed by two researchers after independently screening the literature, extracting information and evaluating quality.

Results  A total of 17 studies were included, including 1 HTA report, 9 systematic reviews/Meta-analyses, and 8 pharmacoeconomic studies. In terms of efficacy, the clinical response rate, clinical remission rate, mucosal healing rate, endoscopic remission rate and short-term colectomy rate were significantly improved by the treatment of tofacitnib in ulcerative colitis patients (P<0.05）. In terms of safety, tofacitinib was similar to placebo in the incidence of adverse events, serious adverse events, withdrawal from treatment due to adverse events, incidence of serious infections, and overall mortality (P>0.05）, with only a slight increase in the incidence of infections (P<0.05）. In terms of cost-effectiveness, the treatment of moderate-to-severe ulcerative colitis with tofacitinib was cost-effective compared with conventional therapy or other biologics in many countries including China.

Conclusion  Tofacitinib is effective and safe in the treatment of ulcerative colitis, and is significantly cost-effective for moderate-to-severe ulcerative colitis patients. Since it is still an off-label drug used for treating ulcerative colitis in China, the efficacy, safety and cost-effectiveness of tofacitinib need to be further evaluated based on the real-world clinical research and medical background in China in the future.

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