Scientists say that thalidomide, a drug once commonly associated with birth defects, helps children and adolescents with Crohn’s disease.
Experts say that up to 1.2 million people in Europe and one-half million people in the United States have Crohn’s disease, an illness in which there is chronic inflammation of the digestive tract.
Around 25 percent of those with Crohn’s disease are children and teens and these cases tend to be more severe than those that develop after people have reached adulthood. In addition, it is not uncommon for children to either not tolerate treatment very well or to develop resistance to treatment. About 18 percent of all childhood cases will require the use of surgery within five year of when the disease begins.
Thalidomide is a drug which is used to treat inflammatory skin and mucous membrane diseases. It is perhaps more commonly recognized as being a drug which was associated with severe birth defects, like missing limbs, in the early 1960’s. It does, however, have other, safer applications than its previous use for nausea during pregnancy.
Marzia Lazzerini of the Institute for Maternal and Child Health in Trieste, Italy and her research team evaluated the safety and efficacy of one such use when they examined how well thalidomide could treat Crohn’s disease in children. The children studied were those with refractory disease, meaning that they had not responded to other common treatments.
The study, which was carried out between August 2008 and September 2012, included 56 children from six pediatric care centers located in Italy. The children were randomly assigned to receive a daily dose of either the drug thalidomide or an inactive placebo for a period of eight weeks.
The researchers evaluated how well the children responded to thalidomide by calculating their Pediatric Crohn’s Disease Activity Index (PCDAI). What they were looking for was a reduction in PCDAI of greater than or equal to 25 percent or greater than or equal to 75 percent at the four and eight week points, which would be considered to be indicative of clinical remission. Those who did not respond to the placebo treatment were then placed on thalidomide treatment for another eight weeks. Any child who responded to treatment with thalidomide was provided with another 52 weeks of therapy.
The team found that more of the children treated with thalidomide were able to achieve remission than those who received placebo (46.4 percent versus 11.5 percent). Although there was no difference in response rates at four weeks of treatment, the thalidomide group did better at eight weeks. Out of the placebo group who went on to receive thalidomide, 52.4 percent achieved remission of symptoms at eight weeks. Overall, 63.3 percent of those treated with thalidomide achieved remission of symptoms while 65.3 percent achieved at least a 75 percent response to the treatment.
The remission of the thalidomide-treated children’s symptoms lasted on average 181 weeks, compared to only 6.3 weeks for those who received an inactive placebo.
The results of the study appear in the November 27, 2013 issue of JAMA.
By Nancy Schimelpfening