A recent study (M Lazzerini et al. Clin Gastroenterol Hepatol 2017; 15: 1382-9) used data from 2 multicenter trials of 70 children to assess the efficacy of thalidomide in pediatric patients with refractory inflammatory bowel disease (37 with Crohn’s disease, 23 with ulcerative colitis)
Key findings:
- 42 patients (60%) had clinical remission & 45 (64%) had clinical response at week 8
- 38 patients (54%) had clinical remission or response at week 52. 29 of these patients had mucosal healing (no erosions or ulcerations) & 20 patients had histologic healing
- 7 patients dropped out from study prior to 52 weeks due to side effects (n=5) or clinical relapse (n=2)
My take: I have not used thalidomide therapy and remain concerned about long term side effects (eg. peripheral neuropathy). Though, the authors are correct that its safety “may be acceptable compared with the safety of other” treatments, especially if there are few remaining options.
Related blog posts:
- JAMA Thalidomide Study for Crohn’s Disease | gutsandgrowth
- When nothing else is working | gutsandgrowth
- Desperate Measures in Refractory IBD
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