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Published on: May 16, 2025
Guideline review: Tofacitinib for adults with moderately to severely active ulcerative colitis - NICE guidance
Akudo Nwaogu1, Ashley Bond2, Philip J Smith2
1Department of Medicine, Royal London Hospital, Barts Health NHS Trust, London, London, UK.
Abstract:
Tofacitinib is an oral, Janus kinase (JAK) molecule, which selectively inhibits Janus-associated tyrosine kinases JAK1 and JAK3. It has already shown efficacy in the treatment of rheumatoid arthritis and the prevention of organ allograft rejection in kidney transplantation. Two separate phase III placebo-controlled trials, assessing 8-week efficacy of tofacitinib induction for ulcerative colitis (UC), demonstrated superiority when compared with placebo. Tofacitinib also demonstrated robust efficacy versus placebo in the 52-week maintenance component of the same study. Tofacitinib has been recommended by the National Institute for Health and Care Excellence as an effective treatment option for adult patients with moderate to severe UC when conventional therapy or a biological agent cannot be tolerated or the disease has responded inadequately or lost response to treatment. We review the guidelines and provide brief commentary on the post hoc analysis related to lipid increases and thromboembolism risk, which have lead to changes in current therapeutic guidance.
Insights
Tofacitinib effectively treats ulcerative colitis (UC) by inhibiting Janus kinases (JAKs). This review covers its efficacy, guidelines, and safety considerations like lipid increases and thromboembolism risk.
Area of Science:
- Immunology
- Gastroenterology
- Pharmacology
Background:
- Tofacitinib is an oral Janus kinase (JAK) inhibitor targeting JAK1 and JAK3.
- It has demonstrated efficacy in rheumatoid arthritis and kidney transplant rejection prevention.
- Ulcerative colitis (UC) is a chronic inflammatory bowel disease requiring effective treatment options.
Purpose of the Study:
- To review the efficacy of tofacitinib in treating moderate to severe ulcerative colitis (UC).
- To discuss current therapeutic guidelines and safety considerations for tofacitinib in UC management.
- To provide commentary on post hoc analyses regarding lipid increases and thromboembolism risk.
Main Methods:
- Review of two separate phase III placebo-controlled trials assessing tofacitinib's 8-week induction and 52-week maintenance efficacy in UC.
- Analysis of National Institute for Health and Care Excellence (NICE) recommendations.
- Examination of post hoc analyses concerning safety outcomes.
Main Results:
- Tofacitinib demonstrated superiority over placebo in both induction and maintenance phases for UC treatment.
- NICE recommends tofacitinib for adult patients with moderate to severe UC unresponsive to conventional or biologic therapies.
- Post hoc analyses identified lipid increases and thromboembolism risks, leading to updated therapeutic guidance.
Conclusions:
- Tofacitinib is an effective oral treatment for moderate to severe UC, particularly when other therapies fail.
- Updated guidance reflects safety considerations, including lipid metabolism and thromboembolic events.
- Continued monitoring and adherence to revised therapeutic recommendations are crucial for optimal patient outcomes.
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