A new randomized, double-blind, placebo-controlled study of 56 children with Crohn's disease suggests pediatric patients treated with thalidomide have better rates of remission and response than those given placebo.
A new randomized, double-blind, placebo-controlled study of 56 children with Crohn’s disease suggests pediatric patients treated with thalidomide have better rates of remission and response than those given placebo.
Marzia Lazzernini, PhD, of the Institute for Maternal and Child Health IRCCS Brio Garofolo in Trieste, Italy, and colleagues reported in JAMA that among trial subjects with active Crohn’s disease in spite of immunosuppressive treatment, those treated with thalidomide had a 4-fold increase in remission compared to those who received placebo (95% confidence interval [CI], 1.2-12.5; P= 0.01).
Thalidomide treatment was associated with a 63.3% clinical remission rate, and 65.3% of patients had a response to treatment of 75% or more, as measured on the Pediatric Crohn’s Disease Activity Index. Patients taking thalidomide also had a much longer duration of remission at 181.1 weeks, versus 5.3 weeks with placebo (P< 0.001).
All patients were followed for 8 weeks, though patients in the placebo group who neither entered clinical remission nor experienced reduction from the baseline activity index score of 75% or more then were monitored for an additional 8 weeks in an open-label extension arm. Thalidomide responders were also followed prospectively for at least an additional 52 weeks in order to study long-term drug efficacy and adverse events.
According to the researchers, there were 9 severe adverse events in total, including peripheral neuropathy and one acute neurologic event.
The authors noted that the children stopped steroid therapy by week 16 and the dose of thalidomide was reduced over the long-term follow-up without a change in efficacy.