Sirolimus may be an effective rescue treatment option for children with refractory inflammatory bowel disease, according to Dr. Mohamed Mutalib and his associates.
In a retrospective analysis of 11 ulcerative colitis (UC) and 3 Crohn’s disease patients treated with sirolimus, 5 of the UC patients and all of the Crohn’s disease patients achieved clinical remission. An additional 2 UC patients achieved clinical response. The remaining 4 UC patients did not respond to sirolimus treatment.
Mucosal healing was achieved in 5 of 11 UC patients and 2 of 3 Crohn’s patients. Clinical response to treatment occurred at least 2 weeks after treatment was started. The only significant side effect reported was minor gastrointestinal distress.
“Our data provide compelling evidence that sirolimus is effective as rescue therapy in a subgroup of children with severe [inflammatory bowel disease] refractory to conventional therapies by inducing both clinical remission and mucosal healing. However, randomized placebo-controlled studies are warranted to extend our encouraging initial findings,” the investigators concluded.
Find the full study in the Journal of Crohn’s and Colitis (doi: 10.1016/j.crohns.2014.08.014).