Thalidomide Is Good for Crohn's Disease in Children
You may have heard that thalidomide use among pregnant women to relieve morning sickness and insomnia was linked to more than 10,000 children being born with birth defects in the late 1950s and early 1960s. Now thalidomide has been found to have a beneficial use among children who have Crohn’s disease.
Crohn’s disease in children
Crohn’s disease is a chronic inflammatory disease that affects the intestinal tract. It is believed to be caused by an interaction between genetic factors, environmental influences, and some type of trigger that sets off the cascade of symptoms.
About 25 percent of the half million cases of the disease that are diagnosed every year in the United States are among individuals younger than 20 years of age. About 30 percent of them have a family history of the disease.
Along with the symptoms of diarrhea, abdominal pain, and rectal bleeding seen in Crohn’s disease patients of all ages, children can experience unique problems such as malnutrition, delay of puberty, loss of bone minerals, and failure to grow properly.
Thalidomide and Crohn’s disease
In a new Italian study published in the Journal of the American Medical Association, the experts studied 54 children who had moderate to severe Crohn’s disease and who had failed to respond to other treatments. During the eight-week study, 28 children were given thalidomide and 26 took a placebo daily.
The study was different in that any of the children who did not improve while taking placebo for eight weeks were then given thalidomide for eight weeks. In addition, any of the 28 children in the treatment group who responded favorably to thalidomide were continued on the drug for at least 52 weeks.
Here’s what the authors discovered:
- Participants who took thalidomide were more likely to go into remission and remission was more likely to last longer than remission achieved by patients taking a placebo
- After eight weeks, more than 46 percent of children who took thalidomide achieved remission compared with only 11 percent of those in the placebo group
- Remission lasted an average of 181 weeks in the thalidomide group compared to 6.3 weeks in the placebo group
- Side effects were not common, and the most frequent one was strange sensations in the legs or arms that disappeared when treatment was stopped
- Thalidomide’s strong sedative impact can be reduced if the drug is taken at night
While thalidomide may be beneficial for children and adolescents who have Crohn’s disease, the drug needs to be discontinued when men and women reach reproductive age. The drug can cause birth defects whether it is taken by women or men as thalidomide can have a negative effect on sperm quality.
Current treatments for Crohn’s disease in children
One reality of Crohn’s disease in children is that it tends to be more severe than when it develops later in life. In fact, about 20 percent of young people with the disease need to have surgery within five years of developing the disease, according to the study’s authors.
For children who have mild disease, starting treatment usually includes 5-aminosalicylic acid (5-ASA) compounds (e.g., sulfasalazine, mesalamine, olsalazine), nutritional therapy, and antibiotics. For more severe disease or for children who do not respond to this approach, doctors prescribe corticosteroids and immunomodulators such as methotrexate or 5-mercaptopurine.
If these attempts are not successful, healthcare providers may turn to biologic therapy, which works by interfering with inflammation. Four biologic drugs have been approved for treatment of Crohn’s disease in adults, and all of them must be given by infusion or injection: adalimumab (Humira), certolizumab (Cimzia), infliximab (Remicade, approved for children ages 6 and older who have ulcerative colitis), and natalizumab (Tysabri).
All of the drugs that can be prescribed for children who have Crohn’s disease have significant side effects. Surgery also is an option.
Further research is needed to verify the findings of this new study. If those results are positive, healthcare providers may then have the option of prescribing thalidomide for treatment of Crohn’s disease in children and adolescents who have not responded to other therapies.
Lazzerini M et al. Effect of thalidomide on clinical remission in children and adolescents with refractory Crohn disease: a randomized clinical trial. Journal of the American Medical Association 2013 Nov 27; 310(20): 2164-73
Crohn's & Colitis Foundation of America