Research Highlights: Thalidomide May Increase Clinical Remission Rates in Children with Pediatric-onset Crohn Disease

Thalidomide May Increase Clinical Remission Rates in Children with Pediatric-onset Crohn Disease

The Journal of the American Medical Association (JAMA) recently featured a study indicating that the drug thalidomide may be an effective treatment for pediatric-onset Crohn Disease. Currently, pediatric-onset Crohn Disease is far more difficult to treat than adult-onset Crohn Disease, as it is more aggressive and is resistant to most preventative drugs. This lack of effective treatment can irreparably harm children afflicted with the disease. This study was thus an important preliminary step in finding new treatment options.
The study looked at 56 children who had been diagnosed with active Crohn disease and whose conditions had not improved after previous treatments. These children were located at six different pediatric clinics in Italy. Investigators randomly assigned children to either a thalidomide treatment or a placebo in a double-blind trial. During the first eight weeks of the trial, 46.4% of the children receiving thalidomide showed clinical remission of Crohn disease, while only 11.5% of children receiving a placebo showed any clinical remission. Some of the children in the placebo group who were not in remission received thalidomide later in the study and 52.4% of this new group also exhibited remission of the disease. In total, experimenters saw clinical remission in 63.3% of the children treated with thalidomide, which was far greater than the initial remission rate (11.5%) of the placebo group.
Furthermore, follow-up studies revealed that children receiving thalidomide experienced much longer periods of clinical remission than the children taking placebos. For children in the thalidomide group, the average remission time was 181.1 weeks, while the average remission time was only 6.3 weeks for children in the placebo group.
Overall, treatment with thalidomide resulted in much greater remission rates of Crohn Disease as well as substantially longer periods of remission. The results of this study thus strongly suggest that thalidomide could be an effective treatment for the disease. Further trials are needed to determine the true efficacy of the drug in treating Pediatric–onset Crohn Disease. For now, however, thalidomide is a promising potential remedy for a disease with few currently effective treatments.

JAMA. 2013;310(20):2164-2173


Caroline Russell-Troutman is the 2013-2014 Research Highlights Editor