Thalidomide may be effective in refractory pediatric Crohn’s disease

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1. After 8 weeks of treatment, thalidomide demonstrated improved rates of clinical remission as compared to placebo.

2. The mean duration of the remission induced by thalidomide was 181 weeks versus the 6 weeks noted in the placebo group.

Evidence Rating Level: 1 (Excellent)

Study Rundown: About a quarter of adult Crohn’s disease patients report symptoms during childhood. Such pediatric-onset of the disease is marked by both a high severity of symptoms and recalcitrance to treatment options. While observational studies on the use of thalidomide, an anti-TNF alpha agent, in Crohn’s disease have reported encouraging results, the present study represents the first randomized controlled trial assessing its efficacy in inducing clinical remission in the pediatric population. The promising findings of this study with regards to remission induction and maintenance highlight the potential utility of thalidomide use in this population. Although the incidence of adverse effects in this study was felt to be acceptable compared to currently utilized immunosuppressant therapy, the study was underpowered to detect rare adverse effects and a more thorough assessment of the safety profile of thalidomide would be helpful prior to its implementation routine clinical care.

Click to read the study, published today in JAMA

Relevant Reading: Specificities of inflammatory bowel disease in childhood

In-Depth [randomized controlled trial]: In this double-blind, placebo-controlled, randomized clinical trial, researchers assembled a cohort of 56 children with active Crohn’s disease refractory to immunosuppression across six pediatric tertiary control centers. Twenty-eight of these children were randomized to receive daily thalidomide while 26 were given daily placebo pills. The Pediatric Crohn Disease Activity Index (PCDAI) was utilized to measure the disease activity at enrollment and track its progression throughout the trial. Clinical remission was assessed at week 8 by a PCDAI score of 10 or less and this was seen in 46.4% of thalidomide recipients versus 11.5% in controls (95%CI, 1.2-12.5). Mean duration of remission was 181.1 weeks (95%CI, 1.4-217.76) in the experimental group and 6.3 weeks (95%CI, 3.51-9.15) in placebo. Of note, peripheral neuropathy was noted to be the most frequent severe adverse event with an incidence noted of 2.1 per 1000 patient-weeks (95%CI, 1.1-4.1).

By Priyanka Vedak and Rif Rahman

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