HealthDay News — Thalidomide is associated with improved clinical remission at eight weeks of treatment for children with refractory Crohn’s disease, according to researchers.
In a multicenter, placebo-controlled study involving children with active Crohn’s disease who failed immunosuppressive treatment, 46.4% of children assigned to thalidomide achieved remission compared with 11.5% of the placebo group (risk ratio 4.0; 95% CI: 1.2-12.5; P =0.01), Marzia Lazzerini, PhD, from the Institute for Maternal and Child Health IRCCS “Burlo Garofolo” in Trieste, Italy, and colleagues reported in the Journal of the American Medical Association.
They randomly assigned 28 children to thalidomide and 26 to placebo once daily for eight weeks. Non-responders to placebo were given thalidomide for an additional eight weeks in an open-label extension.
At four weeks, the responses were not different, but greater improvement was seen at eight weeks in the thalidomide group (75% response: 46.4% vs. 11.5%; 25% response: 64.2% vs. 30.8%; both P=0.01). More than half (52.4%) of the non-responders who received thalidomide subsequently reached remission at week eight.
Overall, 65.3% of children achieved 75% response and 63.3% achieved clinical remission. The mean remission duration was 181.1 weeks in the thalidomide group versus 6.3 weeks in the placebo group (P<0.001).
Cumulative incidence of severe adverse events was 2.1 per 1,000 patient-weeks, with peripheral neuropathy being the most frequent severe adverse event.
“These findings require replication to definitively determine clinical utility of this treatment,” the researchers concluded.