What is the evidence for mycophenolate mofetil in patients with IBD refractory to conventional medical therapy
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mycophenolate mofetil is a reasonable option to consider in IBD refractory or intolerant to conventional immunosuppressive therapy (azathioprine/6-mercaptopurine and methotrexate): a 2009 single-center prospective study found it safe, well tolerated, and efficacious for both short- and long-term therapy without dose escalation, and the authors state it can and should be considered in patients who have failed conventional immunosuppression 1. a good response was also seen in some patients who had previously failed biological agents, suggesting a possible role in that subgroup 1. 1
What is known: - MMF demonstrated efficacy in difficult IBD and appeared safe and well tolerated for short- and long-term therapy without need for dose escalation, with no serious adverse events attributed to it. 1 - Findings supported use of MMF in patients refractory or intolerant to conventional therapies such as azathioprine/6-mercaptopurine or methotrexate. 1 - A good response in some patients who previously failed biological agents suggests a possible role for MMF in that subgroup. 1
What is unknown / caveats: - One study reports MMF safe/efficacious with sustained response; earlier reports describe lack of efficacy, late flares, and non-superiority to azathioprine - Some prior studies suggest MMF dose must be increased over time, though this study did not observe that need - Evidence rests on a single 2009 prospective single-center study - Prior efficacy assessed mainly in small uncontrolled case series and a few small randomized trials, with its role described as controversial - Authors call for larger randomized double-blind trials comparing MMF to conventional immunosuppressants - Earlier literature is mixed. Some studies report late flares, need for dose escalation, and non-superiority to azathioprine, so the overall role of MMF remains unsettled.
## References
1. Tan T, Lawrance IC. Use of mycophenolate mofetil in inflammatory bowel disease. World J Gastroenterol. 2009;15(13):1594-9. PMID: 19340901.
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_Draft, generated by the IBDology RAG and not yet clinician-reviewed. Answers are grounded in the retrieved literature listed above; a high faithfulness score means the answer matches its sources, not that the sources are correct._
Reviewer notes