• M E Gerich, J L Yoon, S R Targan, A F Ippoliti, and E A Vasiliauskas.
• Cedars-Sinai Medical Center, Los Angeles, CA, USA.
• Aliment. Pharmacol. Ther. 2015 Mar 1; 41 (5): 429-37.

BackgroundSeveral open-label and retrospective studies have indicated that thalidomide may be beneficial in patients with refractory Crohn's disease (CD).AimTo report our long-term experience with the use of thalidomide for adults with refractory Crohn's disease.MethodsWe conducted a retrospective study of long-term clinical and safety outcomes among adults treated with thalidomide for refractory Crohn's disease. Response was defined as a clinician's assessment of improvement after at least 7 days treatment of one or more of the following: bowel movement frequency, fistula output, rectal bleeding, abdominal pain, extraintestinal manifestations, or well-being. Remission required all of the following: <3 stools/day, no bleeding, abdominal pain or extraintestinal manifestations and increased well-being.ResultsThirty-seven adults with refractory Crohn's disease were treated with thalidomide for a median of 4.4 months and followed up for a median of 58 months. Clinical response and remission rates were 54% and 19%, respectively. About 40% of patients were able to stop steroids. Response rates were higher for those treated with more than 50 mg/day (85%) than for those treated with a maximum of 50 mg/day (40%; P = 0.01). An adverse event occurred in 68% of patients. Approximately one-third of patients (38%) experienced neuropathy.ConclusionsThalidomide appears to be safe and effective in some patients with refractory Crohn's disease. Although side effects may limit long-term use, thalidomide has potential to induce significant clinical responses.© 2014 John Wiley & Sons Ltd.

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