Thalidomide Shows Promise in Children with Refractory Crohn Disease

F. Bruder Stapleton, MD

An 8-week course, followed by maintenance thalidomide, induced clinical remission in 65% of patients.

Crohn disease (CD) during childhood often requires extensive immunosuppressive therapy. In a randomized, multicenter, placebo-controlled trial, researchers examined the effectiveness of oral thalidomide in 56 children (age range, 2–18 years) with refractory CD despite immunotherapy.

After 8 weeks, 46% of thalidomide recipients reached clinical remission versus 11% of placebo recipients (relative risk, 4.0; number needed to treat, 3). In addition, CD acuity index, erythrocyte sedimentation rate, body weight, and global clinical assessment scores were significantly better in the thalidomide group at 8 weeks. Of 21 placebo nonresponders who crossed over to open-label thalidomide, 11 (52%) reached remission after 8 weeks of therapy, resulting in an overall remission rate of 63%. Mean duration of remission after responding to thalidomide was 181 weeks (vs. 6 weeks in the placebo group). Nine severe adverse events required treatment suspension (cumulative incidence, 2 cases per 1000 patient-weeks). Peripheral neuropathy was the most frequent severe adverse event, followed by amenorrhea, bradycardia, and one case of severe migraine or transient ischemic attack. Cumulative incidence of nonserious adverse events was 12 cases per 1000 patient-weeks.

Citation(s):

Lazzerini M et al. Effect of thalidomide on clinical remission in children and adolescents with refractory Crohn disease: A randomized clinical trial. JAMA 2013 Nov 27; 310:2164.