Medication Showed Promise for Kids With Crohn's

Pediatric Crohns disease patients benefited from thalidomide treatment in Italian study

/ Author:  / Reviewed by: Robert Carlson, M.D Beth Bolt, RPh

(RxWiki News) Young children with inflammatory bowel conditions can face an even more unpleasant illness than their adult counterparts. The authors of a new study explored a possible new treatment.

This study looked at children in Italy with an inflammatory bowel condition called Crohn's disease and the results of treatment with a medication called thalidomide.

The researchers found that young patients treated with thalidomide were more likely to acheive remission from the condition than those who didn't receive the medication.

Remission means that symptoms were greatly reduced or eliminated.

"Talk to your doctor about any chronic digestive issues."

This study, led by Marzia Lazzerini, PhD, of the Institute for Maternal and Child Health IRCCS Burlo Garofolo in Trieste, Italy, focused on children with Crohn's disease.

According to the Crohn's and Colitis Foundation of America (CCFA), Crohn's disease is chronic inflammation of the gastrointestinal tract. Symptoms of the condition can include diarrhea, bleeding, abdominal pain and constipation.

"Pediatric-onset Crohn disease is more aggressive than adult-onset disease, has high rates of resistance to existing drugs, and can lead to permanent impairments," explained Dr. Lazzerini and colleagues.

These researchers focused on a specific medication, thalidomide (brand name Thalomid), which they explained has been used to treat a number of inflammatory conditions, and has shown encouraging results in studies of adult Crohn's patients.

Thalidomide was taken off the markets around the world after problems with birth defects in pregnant women during the 1950s and 1960s. The medication was later approved by the US Food and Drug Administration (FDA) in 1998 for treating an inflammatory complication of the skin condition leprosy. In 2006, the FDA also approved thalidomide (in combination with another medication) for the treatment of certain cases of multiple myeloma, a cancer of the plasma cells found within the bone marrow.

It is important to note that this medication is not currently approved for the treatment of pediatric Crohn's disease in the US. 

Dr. Lazzerini and colleagues followed 56 children between the ages of 2 and 18 years old with active Crohn's disease. The children were identified at six different care centers in Italy, and the study took place between August 2008 and September 2012.

Patients were given either thalidomide (the dose depended on the child's weight) or a placebo (an inactive treatment) once a day for eight weeks.

After the eight weeks were over, those who didn't respond to the placebo treatment were given thalidomide for an additional eight weeks.

Those who showed a response to the thalidomide treatment continued to receive the medication for another 52 weeks.

The researchers determined if patients were in remission from Crohn's disease. This was measured using the Pediatric Crohn Disease Activity Index (PCDAI).

During the first eight weeks, 28 children were given the thalidomide and 26 were given a placebo. Of the thalidomide group, 13 (46.4 percent) achieved remission of Crohn's, compared to three children from the placebo group (11.5 percent).

A total of 21 patients from the original placebo group were then given thalidomide for another eight weeks. During this time, 11 (52.4 percent) achieved remission.

The researchers reported that the average length of remission was 181.1 weeks for the thalidomide group, compared to 6.3 weeks from the placebo group.

A total of nine severe medical events occurred throughout the course of the study. Peripheral neuropathy, or damage to nerves in the peripheral nervous system, was the most commonly seen severe event.

This study involved a small sample size and more research is needed to confirm the findings.

This research will be published November 27 in JAMA, the journal of the American Medical Association. Funding was provided by the Italian Medicines Agency and the medication used in the study was provided by the medication's producers, Pharmion and Celgene.

Review Date: 
November 22, 2013
Last Updated:
December 30, 2013